The FDA (Food and Drug Administration) Drug Approval Process Training Course: Procedures for Submission of INDs, NDAs, ANDAs and 505(b)(2) (ONLINE EVENT: June 11-12, 2025)
DUBLIN--(BUSINESS WIRE)--May 20, 2025--
The 'The FDA (Food and Drug Administration) Drug Approval Process Training Course' training has been added to ResearchAndMarkets.com's offering.
This programme will be especially beneficial to those responsible for preparing US registration documents (INDs,NDAs, Biologics License Applications, etc), regulatory affairs personnel, lawyers and others responsible for advising companies on strategies for developing new drugs for the US market.
The US is the largest market globally for pharmaceutical sales, so having a good understanding of FDA procedures is vital for those submitting in this challenging region. This course will help clarify the US regulatory process, giving you a practical insight into FDA requirements and ensuring that you are fully up to date with all the latest developments.
The comprehensive programme will cover procedures for submission of INDs, NDAs, ANDAs and 505(b)(2), provide a useful insight into the organisation and structure of the FDA and its review processes, and highlight recent changes. The course will emphasise areas of interest to innovative manufacturers, but will also deal with issues relating to generic and over-the-counter drugs.
There will be ample opportunity for discussion with our expert trainer as well as other delegates and a practical workshop session to optimise learning.
Benefits of attending:
Certifications:
Key Topics Covered:
Day 1
Pharmaceutical Medicine and Documents
FDA History and Organisation
Legal Basis (Patent Exclusivity PDUFA, GDUFA, BsUFA)
Legal Basis Continued
Application and Submissions Types
Refusal to File
Investigational New Drug (IND) Applications
Getting Products to the Market Faster
FDA Meetings and Documentation
CTD Content - Setting the Scene
CTD Content - M1
Day 2
CTD Content - M3 and Corresponding M2
CTD Content - M4 and Corresponding M2
CTD Content - M5 and Corresponding M2
Submission Format and Methods
US Amendment Procedures
High Level Comparison US vs EU
Case Study
For more information about this training visit https://www.researchandmarkets.com/r/jjhwpo
About ResearchAndMarkets.com
ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.
View source version on businesswire.com:https://www.businesswire.com/news/home/20250520108035/en/
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KEYWORD:
INDUSTRY KEYWORD: FDA PHARMACEUTICAL HEALTH
SOURCE: Research and Markets
Copyright Business Wire 2025.
PUB: 05/20/2025 08:25 AM/DISC: 05/20/2025 08:24 AM
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The use of companion diagnostics helps guide clinicians in making treatment decisions that can lead to better patient outcomes. 'Following our presentation of positive, late-breaking data from our first-in-class mutCALR-targeted antibody at EHA, we are excited to announce this partnership with QIAGEN, which will facilitate CALR testing for patients with MPNs on a global basis. The development of companion diagnostics for mutCALR, coupled with the potential for new medicines to selectively target disease-initiating cells, is a critical step toward changing the course of disease in patients with ET and MF,' said Pablo J. Cagnoni, M.D., President and Head of Research and Development, Incyte. 'As a partner, QIAGEN has the proven expertise in companion diagnostics development and approvals needed to support our ongoing work and commitment to transforming the treatment of patients with CALR-mutant MPNs.' 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Associated Press
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The event will be webcast live under the investor relations section of Sarepta's website at: and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. 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Words such as 'believe,' 'anticipate,' 'plan,' 'expect,' 'will,' 'may,' 'intend,' 'prepare,' 'look,' 'potential,' 'possible' and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, clinical trials, ELEVIDYS, the potential benefits of an enhanced immunosuppression regimen in dosing in non-ambulatory patients, and expected plans and milestones, including providing additional updates as appropriate and engaging with regulators on an enhanced immunosuppressive regimen for dosing in non-ambulatory patients. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading 'Risk Factors' in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to source version on CONTACT: Investor Contact: Ian Estepan 617-274-4052 [email protected] Contacts: Tracy Sorrentino 617-301-8566 [email protected] Hoeger 617-710-3898 [email protected] KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS INDUSTRY KEYWORD: INFECTIOUS DISEASES HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Sarepta Therapeutics, Inc. Copyright Business Wire 2025. PUB: 06/15/2025 01:00 AM/DISC: 06/15/2025 01:01 AM
Business Wire
6 hours ago
- Business Wire
Positive Late-Breaking Data for Incyte's First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025
WILMINGTON, Del.--(BUSINESS WIRE)--Incyte (Nasdaq:INCY) today announced the first clinical data from two studies evaluating the safety, tolerability and efficacy of INCA033989, a novel, first in class, Incyte-discovered, targeted monoclonal antibody in patients with mutant calreticulin (mutCALR)-expressing myeloproliferative neoplasms (MPNs). These data – featured today in the Late-Breaking Oral Session (#LB4002) at the European Hematology Association 2025 (EHA2025) Congress in Milan, Italy – focus on the dose escalation portion of the studies in patients with high risk essential thrombocythemia (ET) who are resistant/intolerant to prior cytoreductive therapy. 'These findings, and the further development of INCA033989, offer the potential to significantly transform the treatment of patients with CALR-mutant MPNs," said Pablo J. Cagnoni, M.D., President, Head of Research and Development, Incyte. Share The studies evaluated the safety and efficacy of INCA033989 in patients with ET as measured by hematologic response and reduction in mutCALR variant allele frequency (VAF). Results as of April 4, 2025, showed rapid and durable normalization of platelet counts across all dose levels, with a trend toward improved responses in higher doses (>400 mg), in patients with ET treated with INCA033989. Notably, 86% of patients at doses 400 mg and above achieved a complete or partial hematologic response, with the majority (82%) of patients achieving complete response. Eighty-nine (89) percent of evaluable patients (34/38) showed a reduction in mutCALR VAF from baseline. A partial molecular response (>50% VAF reduction) was observed in 21% of evaluable patients (8/38) after only 3 cycles of treatment. An exploratory study using single-cell DNA (scDNA) sequencing showed that INCA033989 directly targets and reduces cells carrying mutCALR. This reduction was seen in early blood-forming (CD34-positive) cells and cells in the myeloid-erythroid (ME) lineage. At the same time, there was a clear increase in healthy (wild-type CALR) cells, suggesting that the treatment supports the return of normal blood production. Bone marrow biopsies further confirmed these effects showing fewer megakaryocytes with mutCALR protein and a notable increase in megakaryocytes without mutCALR protein. Together, these findings demonstrate the selectivity of INCA033989, allowing for normalization of healthy hematopoiesis and disease modification. 'The late-breaking data presented today highlight the impact of INCA033989, a novel agent that selectively targets mutant CALR, to inhibit and eliminate cancer-causing cells in patients with essential thrombocythemia (ET), while sparing healthy cells and normalizing healthy blood production," said Pablo J. Cagnoni, M.D., President, Head of Research and Development, Incyte. 'These findings, and the further development of INCA033989, offer the potential to significantly transform the treatment of patients with CALR-mutant myeloproliferative neoplasms (MPNs).' The results (N=49) showed that INCA033989 was well tolerated across all dose cohorts (24 to 2,500 mg), with no dose-limiting toxicities observed. Only one (1) patient discontinued treatment, and only one (1) dose reduction due to treatment-emergent adverse events (TEAEs) was observed. No infusion interruptions due to TEAEs were reported, and a maximum tolerated dose was not reached. Forty-two (42) patients across the dose cohorts reported a TEAE. The most common TEAEs were fatigue (26.5%) and upper respiratory tract infection (20.4%), all of which were Grade ≤2. Thirteen (13) patients had Grade >3 TEAEs, with transient asymptomatic lipase increase as the most common (6%). 'mutCALR is the second most common oncogenic driver of MPNs, yet the therapeutic landscape lacks a targeted agent for mutCALR expressing MPNs. Currently, ET treatments aim to prevent vascular complications and improve symptoms but are limited by toxicity and tolerability issues,' said John Mascarenhas, M.D., Professor of Medicine at the Icahn School of Medicine at Mt. Sinai and Director, Center of Excellence for Blood Cancers and Myeloid Disorders, The Tisch Cancer Institute. 'These data support the hypothesis that INCA033989 has the potential not only to normalize platelet counts and provide rapid and durable hematologic responses – but to induce molecular responses, which could potentially change the natural history of the disease.' Additional data from the INCA033989 study in patients with myelofibrosis will be submitted for presentation at a future medical meeting. Discussions with regulatory authorities are planned with the goal to initiate a Phase 3 study by early 2026. More information regarding the EHA2025 Congress and the data from Incyte's hematology/oncology portfolio being featured at the meeting can be found on the EHA website: Incyte Conference Call and Webcast Incyte will host an in-person analyst and investor event on Sunday, June 15, 2025, from 6:00 - 7:30 a.m. ET (12:00 - 1:30 p.m. CEST) to discuss key mutCALR data presented at EHA. The event will be webcasted and can be accessed via the Events and Presentations tab of the Investor section of and it will be available for replay for 30 days. About Myeloproliferative Neoplasms Myeloproliferative neoplasms (MPNs) are a closely related group of blood cancers in which the bone marrow functions abnormally. The bone marrow is where the body's blood cells are made. MPNs are progressive blood cancers that can strike anyone at any age, but they are more common in older adults. Estimates of the prevalence of MPNs vary, but analysis of claims data suggests there may be as many as 200,000 people in the U.S. living with the most prevalent MPNs: myelofibrosis, polycythemia vera or essential thrombocythemia (ET). 1 About Mutations in Calreticulin (mutCALR) Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein folding. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs), 2 a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells. 3,4 Among two types of MPNs, essential thrombocythemia (ET) and myelofibrosis (MF), mutCALR drives 25-35% of all cases. 2,3 There are approximately 60,000 patients in the U.S. and Europe with mutCALR positive ET. 5 Incyte is at the forefront of developing novel therapies for patients with mutCALR ET or MF that target only malignant cells, sparing normal cells, including INCA033989, a first-in-class, mutCALR-specific therapy. About the INCA033989 Trial Program The clinical trial program for INCA033989 includes two multicenter, open-label Phase 1 studies, INCA33989-101 (NCT05936359) and INCA33989-102 (NCT06034002), enrolling ~225 patients outside of the U.S. and ~140 patients in the U.S., respectively. The studies are evaluating the safety, tolerability, dose-limiting toxicity (DLT) and maximum tolerated dose (MTD) and/or recommended dose(s) for expansion (RDE) of INCA033989 administered as a monotherapy or in combination with ruxolitinib in patients with myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET) and myelofibrosis (MF). The intent of Part 1A (dose escalation) is to identify the MTD and/or the RDE of INCA033989 among patients with MF and ET. In Part 1A INCA033989 is administered intravenously every two weeks at a protocol defined dose ranging from 24 mg. to 2,500 mg. In Part 1B (dose expansion), INCA033989 is administered at the RDE(s) identified during Part 1A. The primary endpoint of the studies focuses on safety and tolerability as measured by: the number of participants with DLTs up to 28 days, the number of participants with treatment-emergent adverse events (TEAEs) up to 3 years and 60 days, and the number of participants with TEAEs leading to dose modification or discontinuation up to 3 years and 60 days. Secondary endpoints include response rates, mean change of ET total symptom score from baseline, percentage of MF patients achieving spleen volume reduction, MF patient anemia response, mean change in disease-related allele burden and various pharmacokinetics measures up to 3 years and 60 days. For more information on the study, please visit: and About Incyte A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia. For additional information on Incyte, please visit or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube. Forward-Looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the presentation of data for Incyte's anti-mutCALR monoclonal antibody (INCA033989), the potential this monoclonal antibody offers for patients, and expectations regarding ongoing and future clinical trials contain predictions, estimates, and other forward-looking statements. These forward-looking statements are based on Incyte's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; determinations made by the FDA, EMA and other regulatory agencies; Incyte's dependence on its relationships with and changes in the plans of its collaboration partners; the efficacy or safety of Incyte's products and the products of Incyte's collaboration partners; the acceptance of Incyte's products and the products of Incyte's collaboration partners in the marketplace; market competition; unexpected variations in the demand for Incyte's products and the products of Incyte's collaboration partners; the effects of announced or unexpected price regulation or limitations on reimbursement or coverage for Incyte's products and the products of Incyte's collaboration partners; sales, marketing, manufacturing and distribution requirements, including Incyte's and its collaboration partners' ability to successfully commercialize and build commercial infrastructure for newly approved products and any additional products that become approved; greater than expected expenses, including expenses relating to litigation or strategic activities; variations in foreign currency exchange rates; and other risks detailed in Incyte's reports filed with the Securities and Exchange Commission, including its annual report on form 10-K and our quarterly report on Form 10-Q for the quarter ended March 31, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements.
