Rare diseases
Biohaven has suffered a few setbacks in recent months, including an FDA rejection and a missed $150 million benchmark payment, but CEO Vlad Coric looked for the brighter side at JPM, specifically emphasizing a serendipitous discovery that could get the company in the obesity game.
Following rusfertide’s triumphant Phase III trial last year, Protagonist must decide how involved to be in future development. Hundreds of millions of dollars are on the line.
After a cacophony of troubles hit the RNA editing biotech last fall, CEO Ram Aiyar is in San Francisco to develop partnerships, pitch the potential of its new AATD program and find more money to keep the dream alive.
Target action dates for drugs sponsored by Sanofi, Boehringer Ingelheim and Disc Medicine have also been pushed back despite assurances of swift reviews under the FDA’s new Commissioner’s National Priority Voucher program.
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
Acadia Pharma’s Catherine Owen Adams is one of the founders of a group of small- to mid-cap biotechs advocating against a ‘peanut butter blanket’ approach to drug pricing for small companies.
The FDA previously rejected Zycubo for Menkes disease in October last year, citing issues with the drug’s manufacturing facility.
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
After greenlighting 56 novel therapeutics in 2025, four notable applications continue to await the agency’s action after being delayed from the fourth quarter last year.
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D interest as JPM26 kicks off.
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