Rare diseases
After winning a surprise approval for its hereditary angioedema drug Ekterly, KalVista is confident the oral offering will capture the lion’s share of the market for on-demand use.
A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the House is set to vote on later this week.
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.
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