Rare diseases
Vertex Pharmaceuticals’ three cystic fibrosis drugs—Kaftrio/Trikafta, Symkevi and Orkambi—will now be available through England’s National Health Service, following the signing of a long-term reimbursement agreement.
With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year.
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated virus gene therapies to more rare disease patients.
With a total revenue of nearly $2.7 billion, Vertex exceeded analyst estimates in the first quarter, bolstered by strong uptake of its cystic fibrosis products.
Phase II data indicate Larimar Therapeutics’ injectable therapy nomlabofusp could go head-to-head in the market with Biogen’s Skyclarys.
While Pfizer has ended one of its two Phase III studies for inclacumab in sickle cell disease, the company is still eyeing an approval for the antibody in the inherited blood disorder by 2026.
The U.K. National Institute for Health and Care Excellence on Thursday recommended against funding Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy Casgevy unless uncertainties can be cleared up.
Vertex and CRISPR Therapeutics are setting up treatment centers for patients with beta thalassemia and sickle cell disease to compete with bluebird’s established infrastructure.
Sionna Therapeutics on Wednesday announced it raised another round of funding as the company looks to challenge Vertex Pharmaceuticals’ dominant cystic fibrosis business.
Despite recent investment and breakthroughs for rare diseases such as Friedreich’s ataxia and progeria, experts say helping patients with rare diseases will require cross-sector support.
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