Oct. 30 Company Quick Takes: Clinical hold for Zolgensma; plus Biogen-Alkermes, Roche’s satralizumab, Pear-Ironwood

Partial hold for Novartis’ SMA gene therapy
FDA placed a partial clinical hold on studies of intrathecally delivered Zolgensma onasemnogene abeparvovec-xioi from Novartis AG (NYSE:NVS; SIX:NOVN) to treat spinal muscular atrophy after observing dorsal root ganglia mononuclear cell inflammation in a preclinical study. The hold affects the top dose cohort in the Phase I STRONG trial of Zolgensma in patients with SMA type 2 aged up to 5 years old; it does not affect the marketed therapy or studies using the intravenously administrated version. RegenxBio Inc. (NASDAQ:RGNX), whose vector technology is used in Zolgensma, shed $4.69 (12%) to $34.54 Wednesday. The hold is unrelated to this summer’s data integrity scandal concerning the drug (see “Timeline of a Debacle”).

Biogen, Alkermes get full FDA approval for MS drug
Biogen Inc. (NASDAQ:BIIB) and Alkermes plc (NASDAQ:ALKS) received full FDA approval for Vumerity diroximel fumarate to treat relapsing multiple sclerosis. FDA granted tentative approval to the product two weeks ago contingent upon the expiration of an undisclosed patent. The full approval triggers a $150 million milestone payment from Biogen to Alkermes, which is eligible for royalties in the mid-teens (see “Alkermes-Biogen MS Therapy Nears Final FDA Approval”). ...