Latest news with #OrphanDrugDesignation
Business Standard
7 days ago
- Health
- Business Standard
Zydus Lifesciences' Usnoflast receives USFDA fast track designation for ALS Treatment
Zydus Lifesciences announced that the U.S. Food and Drug Administration (USFDA) has granted Fast Track Designation to Usnoflast (ZYIL1), a novel oral NLRP3 inhibitor, for the treatment of Amyotrophic Lateral Sclerosis (ALS). The fast track designation is designed to facilitate the development and expedite the review of drugs that treat serious conditions and address unmet medical needs. Drugs granted this designation are eligible for benefits such as accelerated approval, priority review, and more frequent interactions with the USFDA during the drug development process. The company stated that people living with ALS have an average survival of approximately two to five years from diagnosis, with most ALS patients dying from respiratory failure. ALS patients experience neuroinflammation and rapid neurodegeneration. Axonal neurodegeneration leads to the formation of neurofilaments, which first accumulate in the cerebrospinal fluid (CSF) of ALS patients and then slowly enter the bloodstream. Due to rapid neurodegeneration, steady loss of the ability to move, speak, eat, and eventually breathe, paralysis and death are common outcomes in ALS patients. ALS affects approximately 32,000 people in the U.S., with an average of 5,000 new patients diagnosed every year, according to statistics from the Centers for Disease Control and Prevention (CDC). More than 30,000 people are estimated to be living with ALS in Europe (European Union and United Kingdom), while India has an estimated 75,000 people living with ALS. Usnoflast, a novel chemical entity (NCE), had earlier received Orphan Drug Designation (ODD) from the USFDA, which offers development incentives including tax credits for clinical testing, waiver of certain FDA fees, and a potential seven-year marketing exclusivity upon USFDA approval. Usnoflast (ZYIL1) is a novel oral small molecule NLRP3 inhibitor studied in preclinical models of neuroinflammation, Parkinsons disease, inflammatory bowel disease (IBD), and multiple sclerosis (MS). Zydus has completed a Phase 2(a) randomized, double-blind, placebo-controlled trial in 24 ALS patients across seven sites in India and recently received USFDA approval to initiate a Phase 2(b) randomized, double-blind, placebo-controlled trial in ALS patients. Chairman of Zydus Lifesciences Limited, Pankaj Patel, said, This Fast Track Designation in addition to the previous Orphan Drug Designation granted by the USFDA, underlines the urgent need to develop treatments to address Amyotrophic Lateral Sclerosis (ALS), which is a fatal neurodegenerative disease. Zydus is committed to unlocking new frontiers in neuroscience and develop Usnoflast for patients with ALS. Zydus Lifesciences is a discovery-driven, global life sciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The company reported a 29.62% jump in consolidated net profit of Rs 1,023.5 crore in Q3 FY25 compared with Rs 789.6 crore in Q3 FY24. Revenue from operations increased 16.96% YoY to Rs 5,269.1 crore during the quarter. The counter shed 0.30% to Rs 915.22 on the BSE.
RTÉ News
27-05-2025
- Business
- RTÉ News
Poolbeg Pharma gets FDA support for POLB 001 drug
Biopharmaceutical company Poolbeg Pharma said the US Food and Drug Administration has granted Orphan Drug Designation (ODD) to its POLB 001 product as an oral preventative therapy for Cytokine Release Syndrome (CRS). Cancer immunotherapy-induced CRS is a severe side-effect that occurs in more than 70% of patients and which may lead to multi-organ failure and death. There are currently no approved preventative therapies for CRS. Poolbeg focusses on the development of innovative medicines to address unmet medical needs and its programmes target large addressable markets including, cancer immunotherapy-induced Cytokine Release Syndrome (CRS) and metabolic conditions such as obesity. The company said the FDA grants orphan status to support the development of medicines for rare disorders affecting less than 200,000 people in the US. It provides Poolbeg with clinical development and commercialisation benefits including the potential for a seven-year period of US market exclusivity after regulatory approval of POLB 001, potential waiver exemption of Prescription Drug User Fee Act application fees and the potential for tax credits for qualifying clinical trials. Poolbeg expects the first patient to be dosed in its proposed Phase 2a in the second half of this year, with interim analysis expected in the first half of 2026 and Phase 2a topline data in the second half of 2026. Jeremy Skillington, the chief executive of Poolbeg Pharma, said that POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients' lives. "We were delighted to receive Orphan Drug Designation from the FDA, which is a significant development for Poolbeg and for POLB 001, one that we believe will enhance the commercial appeal for prospective partners and help bring POLB 001 to the market faster," he said. "If approved, we believe POLB 001 has the potential to improve quality of life for patients, reduce pressure on healthcare systems, and expand access to cancer immunotherapies," the CEO added.
Yahoo
21-05-2025
- Business
- Yahoo
Belite Bio Announces FDA Granting of Breakthrough Therapy Designation for Tinlarebant for the Treatment of Stargardt Disease
Designation is based on the pivotal Phase 3 DRAGON trial interim analysis results demonstrating Tinlarebant's efficacy and favorable safety profile Trial completion expected by Q4 2025 (including a three-month follow-up period) Tinlarebant has previously been granted Fast Track and Rare Pediatric Disease Designations in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and the Pioneer Drug Designation in Japan for Stargardt disease SAN DIEGO, May 21, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc. (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tinlarebant for the treatment of Stargardt disease (STGD1) based on the previously reported interim data from the ongoing Phase 3 DRAGON trial. There are currently no approved treatments for STGD1. 'Breakthrough Therapy Designation is an exciting milestone that underscores Tinlarebant's potential to address a serious unmet need for patients with STGD1 — a condition where there are currently no approved therapies,' said Dr. Tom Lin, Chairman and CEO of Belite Bio. 'We remain deeply committed to the Stargardt community and to advancing Tinlarebant as we prepare for the DRAGON study readout expected by the end of this year.' The Breakthrough Therapy Designation is supported by a pre-specified interim analysis of the pivotal, global Phase 3 DRAGON trial data of Tinlarebant in adolescent STGD1 patients. The designation is based on preliminary clinical evidence indicating that a drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. In addition, according to the Data Safety and Monitoring Board (DSMB), Tinlarebant continued to demonstrate a favorable safety profile consistent with prior findings and its mechanism of action. Importantly, visual acuity remained stable in the majority of participants, with mean changes from baseline of less than three letters over the course of the two-year study. 'This granting represents a significant achievement for Belite Bio and serves as validation of a therapeutic approach that directly targets the underlying pathophysiology of STGD1 in order to slow or halt the disease process,' stated Dr. Nathan L. Mata, Belite Bio's Chief Scientific Officer. Dr. Mata noted further, 'Although it has been more than 26 years since the development of the first STGD1 mouse model, it is encouraging to know that the learnings from this model, and the many years spent evaluating the therapeutic benefit of targeting retinol binding protein 4 (RBP4) to reduce the accumulation of cytotoxic byproducts of vitamin A, have advanced our understanding of the disease and have led us closer than ever to the realization of an approved treatment for patients living with STGD1.' 'We are very encouraged by the FDA's decision. STGD1 is a progressive condition that typically begins in adolescence and inevitably leads to legal blindness. People living with STGD1 experience a severe loss of quality of life even though they typically have decades of their lives ahead of them,' said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. 'Breakthrough Therapy Designation reinforces the importance of our work to develop a potential therapy for people who currently have limited options. We look forward to continuing to evaluate Tinlarebant's safety and efficacy as the DRAGON trial progresses.' About FDA Breakthrough Therapy DesignationThe FDA grants Breakthrough Therapy Designation to expedite the development and regulatory review of drugs that are intended for serious or life-threatening conditions. The designation is based on preliminary clinical evidence indicating that a drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Breakthrough Therapy Designation affords all of the benefits of the fast-track program, including eligibility for rolling review and priority review, as well as additional regulatory engagement to facilitate expedited development, with the aim to bring therapies to patients more quickly. About Phase 3 DRAGONThe pivotal Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, global study designed to evaluate the safety and efficacy of Tinlarebant in adolescent patients with Stargardt disease. The trial enrolled 104 subjects across 11 jurisdictions, including the United States, United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization (Tinlarebant:placebo). The primary efficacy endpoint is the growth rate of atrophic lesions, alongside the assessment of safety and tolerability. About TinlarebantTinlarebant is an orally administered, once-a-day tablet intended as an early intervention for maintaining the health and integrity of retinal tissues in Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) patients. Currently, there are no FDA approved treatments for STGD1 and no approved orally administered treatments for GA. Therefore, if approved, Tinlarebant would be a novel oral therapeutic addressing an unmet medical need in both STGD1 and GA. About Belite BioBelite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite's lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on X, Instagram, LinkedIn, and Facebook or visit us at Important Cautions Regarding Forward Looking StatementsThis press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, interim analysis and recommendation from DSMB; Belite Bio's advancement of, and anticipated future activities on preclinical studies, clinical development, regulatory milestones, and commercialization of its product candidates; and any other statements containing the words 'expect', 'hope', 'indicate', 'look forward to', and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio's ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing of potential submission with FDA; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio's drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the 'Risk Factors' section in Belite Bio's filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. Media and Investor Relations Contact:Jennifer Wu ir@ Julie Fallonbelite@ in to access your portfolio
Business Insider
15-05-2025
- Business
- Business Insider
FDA extends PDUFA for Biohaven's Troriluzole new drug application
The company states: 'Biohaven (BHVN) announced that the Division of Neurology 1 within FDA's Office of Neuroscience informed the Company that they are extending the PDUFA date for the troriluzole new drug application for the treatment of spinocerebellar ataxia, SCA, by three months to provide time for a full review of Biohaven's recent submissions related to information requests from the FDA. The Division also informed Biohaven that it is currently planning to hold an advisory committee meeting to discuss the application, but no date has been scheduled. The FDA did not raise any new concerns in the letter. The FDA's decision regarding the NDA is now expected in 4Q 2025. Biohaven previously received Fast-Track, Orphan Drug Designation and Priority Review from the FDA regarding troriluzole for SCA. Priority Review designation is assigned to applications for drugs that would offer a significant improvement over other available treatments for a given disorder or would provide a treatment option where none exists. In the case of SCA, a rare, genetic, neurodegenerative disease, troriluzole would be the first and only FDA-approved treatment for this life-threatening disorder.' Confident Investing Starts Here:
Yahoo
14-05-2025
- Business
- Yahoo
FDA Extends PDUFA Date of Biohaven's Troriluzole NDA for Rare Disease Spinocerebellar Ataxia
- Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. - Troriluzole has been granted Fast-Track, Orphan Drug Designation (ODD) and Priority Review from the FDA. - Troriluzole would be the first and only FDA-approved treatment for SCA, if approved. NEW HAVEN, Conn., May 14, 2025 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), today announced that the Division of Neurology 1 within FDA's Office of Neuroscience informed the Company that they are extending the PDUFA date for the troriluzole new drug application (NDA) for the treatment of spinocerebellar ataxia (SCA) by three months to provide time for a full review of Biohaven's recent submissions related to information requests from the FDA. The Division also informed Biohaven that it is currently planning to hold an advisory committee meeting to discuss the application, but no date has been scheduled. The FDA did not raise any new concerns in the letter. The FDA's decision regarding the NDA is now expected in 4Q 2025. Biohaven previously received Fast-Track, Orphan Drug Designation (ODD) and Priority Review from the FDA regarding troriluzole for SCA. Priority Review designation is assigned to applications for drugs that would offer a significant improvement over other available treatments for a given disorder or would provide a treatment option where none exists. In the case of SCA, a rare, genetic, neurodegenerative disease, troriluzole would be the first and only FDA-approved treatment for this life-threatening disorder. Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven stated, "SCA is a devastating neurodegenerative disease that has affected generations of families and has no current approved therapy. We are committed to bringing the first treatment to patients and families affected by SCA. The clinical data presented in the NDA show a highly favorable benefit-risk profile with troriluzole, a once-daily oral pill, slowing disease progression by 50-70%, as measured by the f-SARA scale, and reducing the risk of falls. We look forward to a meeting with the advisory committee to discuss troriluzole's potential to improve the lives of individuals with SCA. We are especially grateful to the patients, families, and leading neurologists who participated in our studies over an 8-year period and the broader SCA community and patient advocacy groups, including the National Ataxia Foundation, who supported the review of troriluzole as the first potential therapy for SCA." The Company recently announced it has completed the FDA mid-cycle review meeting and regulatory inspections of Biohaven and key clinical research sites for troriluzole in the treatment of SCA. The mid cycle review concluded that there were no previously unidentified major safety concerns, and it does not appear a Risk Evaluation and Mitigation Strategy (REMS) is needed. About Spinocerebellar Ataxia (SCA)Spinocerebellar ataxia is a group of dominantly inherited neurodegenerative disorders characterized by progressive loss of voluntary motor control and atrophy of the cerebellum and brainstem. SCA affects approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom. Patients experience significant morbidity, including impaired gait leading to falls, loss of ambulation and progression to a wheelchair, inability to communicate due to speech impairment, difficulty swallowing, and premature death. While signs and symptoms can appear anytime from childhood to late adulthood, SCA typically presents in early adulthood and progresses over a number of years. Currently, there are no FDA-approved treatments and no cure for SCA. About TroriluzoleTroriluzole is a new chemical entity (NCE) and third-generation novel prodrug that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. The primary mode of action of troriluzole is reducing synaptic levels of glutamate. Troriluzole increases glutamate uptake from the synapse, by augmenting the expression and function of excitatory amino acid transporters located on glial cells that play a key role in clearing glutamate from the synapse. The glutamate modulating activity of troriluzole addresses the widely documented glutamate deregulation that underlies neurodegeneration and Purkinje cell dysfunction in patients with SCA. Troriluzole also has the potential to be developed in a number of other diseases associated with excessive glutamate. More information about troriluzole can be found at the Biohaven's website: About Biohaven Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules and antibody drug conjugates for cancer. For more information, visit Forward-looking Statements This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, including the potential FDA approval and commercialization of troriluzole for SCA, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; including those regarding the potential FDA approval of Troriluzole for SCA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; the potential for Biohaven's product candidates to be first approved therapies; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Investor Contact:Jennifer PorcelliVice President, Investor (201) 248-0741 Media Contact:Mike BeyerSam Brown (312) 961-2502 View original content to download multimedia: SOURCE Biohaven Ltd. Error 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
