Royalty Pharma said it has purchased additional royalties on Roche and PTC Therapeutics’ oral spinal muscular atrophy (SMA) drug for an upfront payment of $1 billion.
Tag Archive for: spinal muscular atrophy
The $75 million deal will leverage Scribe’s CRISPR technologies to develop gene therapies for neurological and neuromuscular disorders. Scribe previously struck deals with Biogen and Sanofi.
Biogen is laying off an unspecified number of employees, according to a article published Monday by the Boston Business Journal.
Biogen Inc. (BIIB.O) Chief Executive Officer Christopher Viehbacher said on Wednesday he is banking on high-profile launches of Alzheimer’s disease treatment Leqembi and depression drug zuranolone, as well as deals, to help the company return to revenue growth.
The European Medicines Agency said it is asking healthcare providers to conduct liver tests on patients treated with Zolgensma, after recent reports of deaths related to the gene therapy.
Last year CDER approved 37 new drugs never before approved or marketed in the U.S., known as “novel” drugs, as noted in its annual New Drug Therapy Approvals report. The agency also approved drugs in new settings, such as for new uses and patient populations.
The spinal muscular atrophy (SMA) community partnered with the non-profit organization Open Style Lab (OSL) to develop a first-of-its-kind runway show, Double Take, that aims to increase authentic disability representation in the creative sphere and spotlight the need for adaptive design in fashion.
Even with the COVID-19 emergency measures, payers still had an eye on the potential flood of gene therapies for rare diseases brewing in the pipeline, and were pondering new ways to pay for them.
