Rare Diseases

Pfizer cited many reasons for ending sales of hemophilia B gene therapy Beqvez, including a lack of patient and doctor interest. The company no longer plans to develop AAV gene therapies.

Nonprofits are finding new ways to address market gaps and develop treatments for rare diseases with little commercial attraction.

Sales of spinal muscular atrophy drug Spinraza had an unexpected US boost in Q4 and Biogen may win US FDA approval for a high dose version this year, but Roche’s Evrysdi was just approved in a new tablet formulation.

HAS, the French health technology assessment body, has issued positive recommendations for several orphan drugs, including for Vyloy, which was provisionally rejected for reimbursement in the UK last year.

A Zydus-Takeda joint venture is expected to supply more raw materials to the Japanese major. Meanwhile, a tie-up with CVS Caremark for the sitagliptin franchise is a boost for Zydus’s US sales while the company focuses R&D efforts on rare diseases like ALS and CAPS

Sionna essentially doubled its cash on hand with its initial public offering to go up against a formidable competitor in cystic fibrosis with novel medicines applicable to a broad population.

CEO Djordje Filipovic tells Scrip that after talks with "multiple other potential partners,” the firm chose the Japanese group for its expertise and marketing capabilities in rare diseases” to be the licensing partner for tadekinig alfa in the US.

Low on cash when Pfizer handed back rights to its hemophilia A gene therapy, Sangamo is talking to partners for its Fabry disease gene therapy to fund its two lead programs through proof-of-concept.

Nine $1bn+ alliances were penned in December, and two exceeded $2bn.

Catalyst saw its share price jump by around 14% after striking what the market termed a favorable patent-litigation settlement agreement with ANDA sponsor Teva over its rare disease drug Firdapse.