FDA & EMA Accept Biogen's Filings for Higher Dose SMA Drug

Regulatory Filings and Drug Development - Biogen announced that the FDA and EMA have accepted its regulatory filings for a higher dose of Spinraza, a blockbuster spinal muscular atrophy (SMA) drug [1] - The new dosing regimen simplifies administration with two initial 50 mg doses spaced 14 days apart, followed by a 28 mg maintenance dose every four months, reducing dosing frequency compared to the current regimen [2] - The regulatory filings are supported by data from the phase II/III DEVOTE study, which showed statistically significant improvement in motor function in infants with SMA treated with a higher dose of Spinraza [3] - A higher dose regimen has the potential to slow neurodegeneration faster through greater reductions in neurofilament as early as day 64, potentially leading to meaningful clinical benefits in infants with symptomatic SMA [4] Spinraza's Market Position and Competition - Spinraza, a key revenue driver for Biogen, was developed jointly with Ionis Pharmaceuticals, with Biogen holding worldwide rights for its development, manufacturing, and commercialization [7] - Spinraza faces competition from Novartis' Zolgensma and Roche/PTC Therapeutics' Evrysdi, both approved for SMA, which has negatively impacted Spinraza's sales in the United States [7][8] - Spinraza's sales declined almost 3% year over year in 2023 and over 13% in the first nine months of 2024 due to increased competitive pressure [8] Biogen's Collaborations and Pipeline - Biogen and Ionis have a collaboration agreement for Qalsody (tofersen), which received FDA accelerated approval in April 2023 for treating adult patients with SOD1-ALS and was also approved in the EU [10] - Biogen and Ionis are collaborating to develop new therapies for neurological diseases, including BIIB094 for Parkinson's disease and BIIB080 for Alzheimer's disease, currently in early to mid-stage studies [11] Stock Performance - Biogen's shares have lost nearly 42% in the past year, compared to the industry's 12% decline [5]