Latest news with #NF1
Yahoo
4 days ago
- Business
- Yahoo
SpringWorks Therapeutics Receives Positive CHMP Opinion for Mirdametinib for the Treatment of Adult and Pediatric Patients with NF1-PN
– If approved, mirdametinib is expected to be the first and only therapy in the European Union with marketing authorization for both adults and children with NF1-PN – – Decision from European Commission expected in the third quarter of 2025 – STAMFORD, Conn., May 23, 2025 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a conditional marketing authorization for mirdametinib, a MEK inhibitor, for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in pediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above. The European Commission (EC) will review the CHMP opinion and is expected to make a final decision regarding the approval in the third quarter of 2025. If approved, mirdametinib will be available in 1 and 2 mg capsules and in a 1 mg dispersible tablet, which dissolves easily in water. 'The positive opinion from the CHMP brings us one step closer to delivering our medicine to both children and adults with NF1-PN in Europe, who we believe are in need of new therapeutic advances,' said Saqib Islam, Chief Executive Officer of SpringWorks. 'Upon approval, we look forward to bringing mirdametinib to appropriate patients in Europe as quickly as possible.' NF1 is a genetic disorder that affects approximately 3 in 10,000 people in the EU, or an estimated 135,000 people.1,2 Patients with NF1 have approximately a 30 to 50% lifetime risk of developing plexiform neurofibromas, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment.3,4 Plexiform neurofibromas can transform into malignant peripheral nerve sheath tumors, an aggressive and potentially fatal disease.5 Surgical removal can be challenging due to the infiltrative tumor growth pattern of plexiform neurofibromas along nerves, and up to approximately 85% of plexiform neurofibromas are considered not amenable to complete resection.6,7,8 'NF1-PN is a genetic disorder that can be highly morbid and unpredictable. It takes a significant physical and emotional toll on patients and their caregivers, and there have been limited treatment options available,' said Ignacio Blanco, MD, PhD, Chairman of the National Reference Center for Adult Patients with Neurofibromatosis at Hospital Universitari Germans Trias i Pujol, Spain. 'Surgical removal of plexiform neurofibromas can be challenging and is often not possible, so if approved, mirdametinib could be an important treatment option for children and adult patients in Europe.' The CHMP opinion was based on the Marketing Authorization Application (MAA) for mirdametinib, which was validated by the EMA in August 2024. The MAA centered on the primary results from the Phase 2b ReNeu trial, which enrolled 114 patients with NF1-PN age 2 years or older (58 adults and 56 pediatric patients). The study met the primary endpoint of confirmed objective response rate (ORR), as assessed by blinded independent central review, demonstrating an ORR of 41% (N= 24/58) in adults and 52% in children (N=29/56). The median best percentage change in target PN volume was -41% (range: -90 to 13%) in adults and -42% (range: -91 to 48%) in children. Among those with a confirmed response, 88% percent of adults and 90% of children had a response of at least 12 months duration, and 50% and 48%, respectively, had a response of at least 24 months duration. Both adults and children also experienced early and sustained significant improvements from baseline in pain and quality of life as assessed across multiple patient-reported outcome tools.9 Mirdametinib demonstrated a manageable safety and tolerability profile. The most common adverse events (>25%) reported in adults receiving mirdametinib were rash, diarrhea, nausea, musculoskeletal pain, vomiting and fatigue. The most common adverse events (>25%) occurring in children were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction and nausea.9 Mirdametinib is approved in the U.S. for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. About the ReNeu TrialReNeu (NCT03962543) is an ongoing, multi-center, open-label, single arm, Phase 2b trial evaluating the efficacy, safety and tolerability of mirdametinib in patients ≥2 years of age with an inoperable NF1-associated PN causing significant morbidity. The study enrolled 114 patients to receive mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose of 4 mg twice daily) without regard to food. Mirdametinib was administered orally in a 3-week on, 1-week off dosing schedule as either a capsule or dispersible tablet. The primary endpoint is confirmed objective response rate (ORR) defined as the proportion of patients with a ≥20% reduction in target tumor volume on consecutive scans during the 24-cycle treatment phase, as measured by MRI and assessed by blinded independent central review. Secondary endpoints include safety and tolerability, duration of response, and changes in patient-reported outcomes from baseline to Cycle 13. The treatment phase of the trial is complete, and results were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Patients who completed the treatment phase were eligible to continue receiving treatment in the optional long-term follow-up portion of the study, which is ongoing. About NF1-PNNeurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene, which encodes for neurofibromin, a key suppressor of the MAPK pathway.10,11 NF1 is the most common form of neurofibromatosis, with an estimated global birth incidence of approximately 1 in 2,500 individuals.3,12 In the EU, NF1 affects approximately 3 in 10,000 people, or an estimated 135,000 people.1,2 The clinical course of NF1 is heterogeneous and manifests in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment.13 Patients with NF1 have an 8 to 15-year mean reduction in their life expectancy compared to the general population.1 NF patients have approximately a 30%-50% lifetime risk of developing plexiform neurofibromas, or PN, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment; in rare cases, NF1-PN may be fatal.3,4,5 NF1-PNs are most often diagnosed in the first two decades of life.3 These tumors can be aggressive and are associated with clinically significant morbidities; typically, they grow more rapidly during childhood.14,15 Surgical removal of these tumors can be challenging due to the infiltrative tumor growth pattern along nerves and can lead to permanent nerve damage and disfigurement.5 Up to approximately 85% of plexiform neurofibromas are considered not amenable to complete resection.6,7,8 About MirdametinibMirdametinib is an oral, small molecule MEK inhibitor approved in the United States for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. The FDA and the European Commission have granted Orphan Drug designation for mirdametinib for the treatment of NF1. About SpringWorks TherapeuticsSpringWorks is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with severe rare diseases and cancer. We developed and are commercializing the first and only FDA-approved medicine for adults with desmoid tumors and the first and only FDA-approved medicine for both adults and children with neurofibromatosis type 1 associated plexiform neurofibromas (NF1-PN). We are also advancing a diverse portfolio of novel targeted therapy product candidates for patients with both solid tumors and hematological cancers. For more information, visit and follow @SpringWorksTx on X, LinkedIn, Facebook, Instagram and YouTube. Forward-Looking Statements This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development and commercialization plans, our preclinical and clinical results, our expectations regarding the timing and results of the EMA's review of our MAA for mirdametinib and our plans to begin its initial launch in the European Union in 2025, our plans to continue to study mirdametinib in pediatric and young adult patients with low-grade gliomas in a Phase 2 study, as well as relating to other future conditions. Words such as, but not limited to, 'look forward to,' 'believe,' 'expect,' 'anticipate,' 'estimate,' 'intend,' 'plan,' 'would,' 'should' and 'could,' and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the fact that topline or interim data from clinical studies may not be predictive of the final or more detailed results of such study or the results of other ongoing or future studies, (ii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iii) the timing of our planned regulatory submissions and interactions, including the timing and outcome of decisions made by the FDA, EMA, and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, (iv) whether EMA or other regulatory authorities will require additional information or further studies, or may fail or refuse to approve or may delay approval of our product candidates, including mirdametinib, (v) our ability to obtain regulatory approval of any of our product candidates or maintain regulatory approvals granted for our products, (vi) our plans to research, discover and develop additional product candidates, (vii) our ability to enter into collaborations for the development of new product candidates and our ability to realize the benefits expected from such collaborations, (viii) our ability to maintain adequate patent protection and successfully enforce patent claims against third parties, (ix) the adequacy of our cash position to fund our operations through any time period indicated herein, (x) our ability to establish manufacturing capabilities, and our collaboration partners' abilities to manufacture our product candidates and scale production, and (xi) our ability to meet any specific milestones set forth herein. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the 'Risk Factors' in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2025 as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings. Contacts: MediaMedia@ InvestorsInvestors@ References Lee TJ, et al. Incidence and prevalence of neurofibromatosis type 1 and 2: a systematic review and meta-analysis. Orphanet J Rare Dis. 2023;18(1):292. doi:10.1186/s13023-023-02911-2. Eurostat. Population and population change statistics. Eurostat Statistics Explained. 2024. Available at: Accessed May 8, 2025. Prada C, Rangwala F, Martin L, et al. Pediatric Plexiform Neurofibromas: Impact on Morbidity and Mortality in Neurofibromatosis Type 1. J Pediatr. 2012;160(3):461-467. doi:10.1016/ Miller DT, et al. Health supervision for children with neurofibromatosis Type 1. Pediatrics. 2019;143(5):e20190660. Kamaludin, Siti Nurhazwani, et al. 'Plexiform neurofibromatosis with peripheral malignant nerve sheath tumor and scoliosis - more surveillance imaging needed?' Radiology case reports vol. 17,7 2388-2393. 6 May. 2022, doi:10.1016/ Needle M, Cnaan A, Dattilo J, et al. Prognostic signs in the surgical management of plexiform neurofibroma: The Children's Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997;131(5):678-682. doi:10.1016/s0022-3476(97)70092-1. Ejerskov, C., Farholt, S., Nielsen, F.S.K. et al. Clinical Characteristics and Management of Children and Adults with Neurofibromatosis Type 1 and Plexiform Neurofibromas in Denmark: A Nationwide Study. Oncol Ther 11, 97–110 (2023). doi:10.1007/s40487-022-00213-4. Wolkenstein, P. et al. (2023) 'French cohort of children and adolescents with neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas: Cassiopea study', European Journal of Medical Genetics, 66(5), p. 104734. doi:10.1016/ Moertel CL, Fisher MJ, Weiss BD, et al. ReNeu: A pivotal, Phase IIb trial of mirdametinib in adults and children with symptomatic neurofibromatosis type 1-associated plexiform neurofibroma. J Clin Oncol. 2024;JCO.24.01034. doi:10.1200/JCO.24.01034. Yap YS, McPherson JR, Ong CK, et al. The NF1 gene revisited - from bench to bedside. Oncotarget. 2014;5(15):5873-5892. doi:10.18632/oncotarget.2194. Rasmussen S, Friedman J. NF1 Gene and Neurofibromatosis 1. Am J Epidemiol. 2000;151(1):33-40. doi:10.1093/ Ferner R. Neurofibromatosis 1 and neurofibromatosis 2: a twenty first century perspective. The Lancet Neurology. 2007;6(4):340-351. doi:10.1016/s1474-4422(07)70075-3. Weiss, Brian D et al. 'NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults with NF1-Related Plexiform Neurofibromas.' Journal of clinical oncology: official journal of the American Society of Clinical Oncology vol. 39,7 (2021):797-806. doi:10.1200/JCO.20.02220. Gross A, Singh G, Akshintala S, et al. Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1. Neuro Oncol. 2018;20(12):1643-1651. doi:10.1093/neuonc/noy067. Nguyen R, Dombi E, Widemann B, et al. Growth dynamics of plexiform neurofibromas: a retrospective cohort study of 201 patients with neurofibromatosis 1. Orphanet J Rare Dis. 2012;7(1):75. doi:10.1186/ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Metro
19-05-2025
- Health
- Metro
Man claims cafe staff refused to serve him due to face tumours
A man with a rare genetic condition has said he was refused service in a cafe after customers stared at him like he was a 'ghost'. 35-year-old Amit Ghose has Neurofibromatosis Type 1, a condition which causes benign tumours to grow on his nerves. Last month, Amit had been visiting his sister Shilu Sen in Wood Green, London, when they went out for a coffee. When he entered a cafe, he says he noticed people were looking at him 'like they'd seen a ghost'. As Amit ordered a drink, he said he was told by the staff that they were 'no longer serving.' 'I went into this coffee shop and it was full of people, mainly men, and they were all just staring at me, like they'd seen a ghost,' he said. 'I went into this coffee shop and it was full of people, mainly men, and they were all just staring at me, like they'd seen a ghost. The barista told me they weren't serving anymore and then just turned and walked away. He added: 'It was not a good feeling, obviously – I felt neglected. It's not very inclusive, it's not acceptable to be subjected to that kind of behaviour.' Amit said he felt disheartened walking out of the shop, but later went to a separate shop, where the staff were 'lovely'. 'It's not all doom and gloom in the world, but it does happen,' he said. Amit, from Birmingham, was born with the rare genetic condition that affects approximately one in 2,500 to 3,000 people worldwide. At the age of 11, his left eye was surgically removed, which led him to wear an eye patch for six months while his prosthetic eye was built. Despite his disability, Amit gives talks in schools about his condition and has written a children's book called 'Born Different'. He said: 'A kid once said to me: 'You don't need a Halloween mask, you've got one for life.' That comment absolutely broke me, I still think about it every Halloween.' After learning to 'accept' his facial difference and with the support of his wife, Piyali, 28, Amit started sharing his story online to help others. He has gained 300,000 followers across his social media platforms over the past two years, and even quit his job as a regional manager at a law firm to pursue a career as a motivational speaker, content creator, and a DEI (Diversity, Equity, and Inclusion) Business Partner. 'People with visible differences are human beings. I want to help other people find validation and security in themselves,' he said. According to the NHS, NF1 is a condition people are born with, but symptoms can develop gradually over many years. The tumours are usually non-cancerous and benign, but can cause symptoms including neurofibromas, birthmarks, freckles in unusual places, or problems with bones, eyes and the nervous system. NF1 is caused by a faulty gene, and there's a 1 in 2 chance a child will develop NF1 if either the mother or father has it. There's currently no cure for the condition, but treatment can involve surgery, medicine to control secondary conditions, physiotherapy and pain management. Around 1 in 3,000 people have the condition, but Charmaine's case is extreme. If you or someone you know is affected by Neurofibromatosis visit for support and further information In 2022, another London cafe defended its decision to effectively bar a blind man with learning disabilities as his guide dog was 'too large and fluffy'. More Trending Staff at the Star Cafe in Sidcup, south-east London, repeatedly turned away Stephen Vallance, 44, his mum Ann and his Alsatian-Labrador cross, Wills, who is neither particularly large nor fluffy. He was told to either sit outside in the cold or leave, despite laws banning businesses from discriminating against guide dog owners. The charity Guide Dogs UK slammed the cafe's 'illegal' actions as 'unacceptable' and 'extremely disappointing'. Get in touch with our news team by emailing us at webnews@ For more stories like this, check our news page. MORE: Man, 26, dies after being stabbed during 'altercation' in north west London MORE: 'Knee knockers' are clogging the London Tube network and commuters are furious MORE: Restaurant tipping rules are quietly changing — and service charges could rise to 20%
Yahoo
14-05-2025
- Business
- Yahoo
Pasithea Therapeutics Announces Initiation of Phase 1/1B Study of PAS-004 in Adult NF1 Patients and Activation of First Clinical Trial Site
-- First patient expected to be dosed during Q2 2025 ---- Trial will evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in both plexiform neurofibromas and cutaneous neurofibromas ---- Starting dose of 4mg tablet QD (once daily) ---- First trial site in Australia. Four additional sites planned for Australia, South Korea, and U.S. –-- Australian R&D Tax Incentive refund of up to 48.5% of eligible study-related costs expected -- MIAMI, May 14, 2025 (GLOBE NEWSWIRE) -- Pasithea Therapeutics Corp. (NASDAQ: KTTA) ('Pasithea' or the 'Company'), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor today announced initiation of its Phase 1/1b open label study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of PAS-004, in adult participants with neurofibromatosis type 1 (NF1) with symptomatic and inoperable, incompletely resected, or recurrent plexiform neurofibromas. The study will also assess preliminary anti-tumor activity and help determine a recommended dose for subsequent Phase 2 trials. Exploratory objectives include assessing the effects of PAS-004 on cutaneous neurofibromas. The first active clinical trial site is the Royal North Shore Hospital in Sydney, Australia, which is expected to begin patient enrollment in Q2 2025. Additional clinical trial sites in Australia, South Korea, and the United States are expected to be opened in the coming months. Pasithea has selected Novotech (Australia) Pty Limited as its clinical research organization (CRO) for this trial. The Company is conducting the study through its wholly owned subsidiary in Australia, Pasithea MacroMEK Pty Ltd, and anticipates eligibility for an Australian R&D Tax Incentive with a cash refund of up to 48.5% of the amount spent annually on eligible R&D activities (trial costs) in Australia. Dr. Rebecca Brown, M.D., Ph.D. a member of Pasithea's Scientific Advisory Board and Associate Professor of Neuro Oncology at The University of Alabama at Birmingham commented, 'I am pleased to have collaborated with the Pasithea team on the design of a comprehensive dose exploration and expansion study to assess the safety and tolerability of PAS-004 in adult NF1 patients. In addition to testing the effects of PAS-004 on plexiform neurofibromas, exploratory endpoints will also examine the effects of PAS-004 on cutaneous neurofibromas. The safety profile observed to date in advanced cancer patients is encouraging, and I look forward to seeing that profile translate to the NF1 population.' Dr. Brown added, 'One of the biggest challenges in treating plexiform neurofibromas associated with NF1 is ensuring that patients remain on MEK inhibitor therapy over the long-term. Real-world data shows that a significant proportion of NF1 patients discontinue treatment due to poor tolerability, including high rates of rash and gastrointestinal side effects. PAS-004 is also given as a once daily dose that offers a more convenient regimen than current FDA-approved therapies that are dosed twice a day and which could improve patient compliance.' Dr. Tiago Reis Marques, Pasithea's Chief Executive Officer, said, 'Following our recent financing, including the exercise of certain warrants, Pasithea is now funded to produce initial interim patient data in NF1. The initiation of this clinical trial in NF1, the initial indication we seek FDA marketing approval for, marks an important milestone for Pasithea and for patients living with NF1-related plexiform neurofibromas. Activating our first clinical trial site underscores our commitment to advancing PAS-004 as a potential best-in-class next-generation MEK inhibitor. We are encouraged by the safety and clinical data observed to date in oncology patients and are optimistic that PAS-004's tolerability profile will extend to the NF1 population. Importantly, our existing cancer data has enabled us to begin the NF1 trial at a higher dose than originally contemplated. In addition, we anticipate meaningful cash rebates of eligible trial costs through the Australian R&D Tax Incentive, further enhancing the efficiency of this program.' About the Phase 1/1b Clinical Trial in Adult NF1 Patients The primary objective of the Phase 1/1b study (NCT06961565) is to evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle in adult NF1 participants with at least one and up to two additional target plexiform neurofibromas (PNs) that are symptomatic and inoperable, incompletely resected, or recurrent. Secondary objectives are (i) to identify the recommended Part B dose ('RPBD') or Maximum Tolerated Dose (MTD) of PAS-004, (ii) to characterize the PK and PD profile of PAS-004, (iii) to evaluate the preliminary efficacy of PAS-004 on target PN volume, (iv) to evaluate the preliminary efficacy of PAS-004 on the size, appearance, and associated symptoms of cutaneous neurofibromas (CNs), and (v) to evaluate the impact of PAS-004 on quality of life ('QOL') and any physical symptoms attributed to the target PN. Experimental objectives are (i) to evaluate the impact of PAS-004 on QOL and any physical symptoms attributed to CNs, (ii) to evaluate the impact of PAS-004 on pain and function attributed to PNs, and (iii) to investigate PAS-004 effects on CN tumor cellular and molecular biology. The trial will be conducted in two parts. In Part A, following a screening period of up to 28 days, up to 24 eligible participants will be enrolled sequentially to receive one of four planned dose levels of PAS-004 tablets (4mg, 8mg, 12 mg, 18mg) in a modified 3+3 design. Part A will identify the recommended RPBD. During Part B, up to 24 eligible participants will be enrolled in parallel to receive one of two planned dose levels of PAS-004 tablets. Participants will be dosed at the RPBD level and at a dose level below the RPBD for up to six continuous 28-day treatment cycles. Part B will identify the recommended phase 2 dose (RP2D). The study is planned to be conducted at five clinical trial sites in Australia, South Korea and the U.S. To learn more about the PAS-004 clinical trial in adults with NF1-associated plexiform neurofibromas, please visit About Pasithea Therapeutics Corp. Pasithea is a clinical-stage biotechnology company focused on the discovery, research and development of innovative treatments for central nervous system (CNS) disorders, RASopathies and MAPK pathway driven tumors. Forward-Looking Statements This press release contains statements that constitute 'forward-looking statements' made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include statements regarding the Company's ongoing Phase 1 clinical trial of PAS-004 in advanced cancer patients, the Company's Phase 1/1b clinical trial of PAS-004 in adult patients with NF1-associated plexiform neurofibromas, and the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD) and preliminary efficacy of PAS-004, as well as all other statements, other than statements of historical fact, regarding the Company's current views and assumptions with respect to future events regarding its business, as well as other statements with respect to the Company's plans, assumptions, expectations, beliefs and objectives, the success of the Company's current and future business strategies, product development, pre-clinical studies, clinical studies, clinical and regulatory timelines, market opportunity, competitive position, business strategies, potential growth opportunities and other statements that are predictive in nature. Forward-looking statements are subject to numerous conditions, many of which are beyond the control of the Company. While the Company believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to the Company on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties, including risks that future clinical trial results may not match results observed to date, may be negative or ambiguous, or may not reach the level of statistical significance required for regulatory approval, as well as other factors set forth in the Company's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q and other filings made with the U.S. Securities and Exchange Commission (SEC). Thus, actual results could be materially different. The Company undertakes no obligation to update these statements whether as a result of new information, future events or otherwise, after the date of this release, except as required by law. Pasithea Therapeutics Contact Patrick GaynesCorporate Communicationspgaynes@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
12-02-2025
- Health
- Yahoo
FDA approves Gomekli as first neurofibromatosis drug for adults
The US Food and Drug Administration (FDA) has approved SpringWorks Therapeutics' Gomekli (mirdametinib) to treat neurofibromatosis type 1 (NF1), a type of rare genetic disorder. Both adult and paediatric patients with NF1 who have symptomatic plexiform neurofibromas (PN) that cannot be surgically removed will be eligible for the treatment. NF1 is a genetic disorder that currently affects approximately 100,000 children and adults in the US. The NF1 gene codes for neurofibromin, a protein that acts as a tumour suppressor. Patients with the genetic disorder have a 30%-50% lifetime risk of developing PN, which are tumours that grow along the peripheral nerve sheath and are difficult to surgically remove. They can cause skin disfigurement, pain and functional impairment. AstraZeneca's Koselugo (selumetinib) became the first drug approved for NF1-PN in the US in 2020, although it is only indicated for paediatric patients aged two years and older. SpringWorks estimates that around 40,000 people in the US, both children and adults, live with NF1-PN. Both Gomekli and Koselugo are kinase inhibitors that target mitogen-activated protein kinase kinases 1 and 2. These enzymes are responsible for cell growth and are overactive in patients with NF1. SpringWorks' drug demonstrated objective response rates of tumour volume reduction in 41% and 52% of adults and children respectively in the Phase 2b ReNeu trial (NCT03962543). Among those who responded to the treatment, 88% of adults and 90% of children had sustained positive effects after a year. Gomekli's safety and tolerability profile is described as 'manageable', with the most common adverse events across adults and children being vomiting, rash, and diarrhoea. Lead trial investigator Dr Christopher Moertel said: 'It was very encouraging in the ReNeu trial to see that Gomekli provided deep and durable responses, with a manageable safety profile that enabled patients to stay on therapy. This approval represents an important advance, especially for adults who previously did not have an approved treatment.' Gomekli is available in capsules or oral suspension and Connecticut-based SpringWorks expects to distribute the treatment in the US within two weeks. The drug is also currently under regulatory review in Europe, with a decision expected later this year. Due to Gomekli's rare paediatric designation, SpringWorks received a priority review voucher upon approval. This voucher allows the company to fast-track a future candidate of its choice through FDA review, meaning a market placement of four months sooner than usual. SpringWorks could also sell the asset for a quicker cash injection – sales of vouchers have reached more than $100m in recent years. The FDA approval comes at a time when German drugmaker Merck KGgA is eyeing SpringWorks as a potential acquisition. Originally reported by Reuters, Merck confirmed it is in advanced discussions with the Gomekli developer though 'no legally binding agreement has been entered into'. If an agreement is reached, the takeover could happen within a few weeks. SpringWorks share price soared by 34% following the disclosure, bringing its market cap to exceed $4bn. "FDA approves Gomekli as first neurofibromatosis drug for adults" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
