Sarepta Therapeutics

Solid Biosciences Inc. received FDA Fast Track designation for SGT-003, a next-generation gene therapy for Duchenne muscular dystrophy (DMD), using AAV-SLB101 capsid. This designation aims to expedite development and review, following encouraging pre-clinical data. The company's stock surged 68.4% post-announcement. A phase I/II study will assess SGT-003's safety and efficacy in pediatric DMD patients.

2023 saw significant progress in DMD treatment with Sarepta Therapeutics’ Elevidys approval, yet it benefits only a small patient group. Challenges remain in therapy's long-term efficacy, safety, and expanding access. Research continues for broader, more effective treatments, emphasizing the need for innovative strategies and precise outcome measures.

Catalyst Pharmaceuticals announced FDA approval for Santhera Pharmaceuticals' DMD treatment, Agamree, for patients aged two and older. The drug, similar to corticosteroids but with fewer side effects, is set for U.S. launch in Q1 2024. Catalyst plans a financial assistance program to enhance drug accessibility.

Capricor Therapeutics aligned with the FDA on the HOPE-3 trial design for CAP-1002, targeting Duchenne muscular dystrophy (DMD) treatment. The trial aims for 58 participants, with topline results expected in late 2024 and a BLA submission in 2025. Positive 24-month data from HOPE-2-OLE study showed CAP-1002's potential benefits in cardiac and skeletal function for DMD patients.

The FDA approved Elevidys, the first gene therapy for Duchenne muscular dystrophy (DMD) in children aged 4-5 with a specific DMD gene mutation. It produces a micro-dystrophin protein to address the genetic cause, differing from symptom-focused treatments. Approval was based on increased protein expression, predicting clinical benefit, with ongoing trials to confirm efficacy.

Sarepta Therapeutics received FDA accelerated approval for Elevidys, a gene therapy for DMD in children aged 4-5, pending confirmatory trial results. Bristol Myers Squibb's Camzyos was approved in the EU for treating obstructive HCM. FibroGen's pamrevlumab failed in a phase III IPF study, leading to discontinuation of related trials. Intercept Pharmaceuticals faced FDA rejection for its NASH treatment, prompting restructuring. MoonLake Immunotherapeutics reported positive phase II results for sonelokimab in treating HS.

Conner Curran's Duchenne muscular dystrophy diagnosis at age 4 initially seemed hopeless, but advances in gene therapy offer new hope. With 50 treatments in development and the first gene therapy potentially FDA-approved soon, the future for Duchenne's patients is changing. Despite challenges, including treatment costs and risks, early intervention and ongoing research aim to significantly improve quality of life and outcomes for affected children.

The pandemic has elevated the role of medical affairs professionals, emphasizing the need for accurate information and staying updated. Key virtual conferences in late 2020 and early 2021 include the Medical Affairs Strategic Summit, Pharmaceutical Regulatory Affairs and IPR Conference, MedComm 3.0, Medical Affairs Leadership Forum, Medical Affairs in Rare Diseases Forum, 20th Medicinal & Pharmaceutical Sciences Congress, MAPS EMEA 2020, Medical Affairs Congress, Pharmacovigilance USA 2021, and Pharma Europe 2021, focusing on strategies, networking, and professional development.

FDA emphasizes the importance of confirmatory trials being underway before granting accelerated approvals, especially in oncology, to ensure clinical benefits. Despite challenges, including delays and withdrawals, the agency seeks to streamline the process and improve clinical trial diversity, advocating for simpler trials and closer collaboration with the National Cancer Institute.

FDA's accelerated approval process allows drugs like Makena to market without proven effectiveness, risking patient safety. Despite required follow-up studies, many drugs remain unverified, with companies delaying research. Critics argue this process exploits patients, offering expensive treatments of questionable benefit.