The agencies announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases.
Moderna is preparing for multiple product launches over the next several years, including RSV and flu vaccines and boosters for COVID-19. It is also aiming mRNA programs at rare diseases.
Attendees will learn barriers and effective strategies to mirroring the patient community make-up in rare disease clinical trials; challenges that rare disease patient organizations are tackling to improve diversity, equity and inclusion (DE&I) in their own communities; and consequences of getting it ‘wrong’ in the clinical stage and beyond.
Xenpozyme is the first approved medication to treat symptoms that are not related to the central nervous system in patients with ASMD, a rare genetic disease that causes premature death.
Blueprint Medicines’ Ayvakit (avapritinib) is headed to the FDA for a new indication after the drug hit the mark in Part 2 of the PIONEER trial for non-advanced systemic mastocytosis.
ANI Pharmaceuticals shares how a data-driven go-to-market approach can help lean teams effectively launch and grow rare diseases brands.
Q&A with Michael Dunn of Genentech
The U.S. Food and Drug Administration has placed Astellas Pharma’s Phase I/II FORTIS trial on clinical hold after one of its study participants experienced a serious adverse event (SAE).
North Carolina-based 9 Meters Biopharma, a clinical-stage company that creates treatments for digestive diseases, is discontinuing its Phase III trial studying larazotide for the treatment of celiac disease due to disappointing interim results.
Pharmaceutical powerhouse AstraZeneca appears to have taken an interest in the company’s London-based partner Mereo BioPharma and may be considering a buyout, according to circulating speculations first reported by The Times UK.