US FDA rejects higher dosage approval for Biogen's genetic disorder medication
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Sep 23 2025
0mins
Source: Reuters
FDA Decision: The U.S. FDA has declined to approve a higher-dose version of Biogen's drug Spinraza for spinal muscular atrophy, requesting updated technical information but not citing deficiencies in clinical data.
Market Competition: Biogen faces increasing competition from Novartis' Zolgensma and Roche's Evrysdi, which offer easier administration and broader accessibility, while the high-dose regimen is already approved in Japan and under review in Europe.
Analyst Views on IONS
Wall Street analysts forecast IONS stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for IONS is 92.67 USD with a low forecast of 65.00 USD and a high forecast of 110.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
14 Analyst Rating
13 Buy
1 Hold
0 Sell
Strong Buy
Current: 81.790
Low
65.00
Averages
92.67
High
110.00
Current: 81.790
Low
65.00
Averages
92.67
High
110.00
About IONS
Ionis Pharmaceuticals, Inc. develops six marketed medicines for serious diseases, including medicines for neurologic and cardiovascular diseases. Its marketed medicines consist of TRYNGOLZA, WAINUA (eplontersen), SPINRAZA (nusinersen), QALSODY (tofersen), TEGSEDI (inotersen) and WAYLIVRA (volanesorsen). TRYNGOLZA reduces triglyceride levels in adults with familial chylomicronemia syndrome (FCS). WAINUA is approved for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults. SPINRAZA is used for the treatment of pediatric and adult patients with spinal muscular atrophy. QALSODY is approved for the treatment of Amyotrophic Lateral Sclerosis in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene, or SOD1-ALS. TEGSEDI is approved for the treatment of ATTRv-PN in adults. WAYLIVRA is approved as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk of acute, potentially fatal pancreatitis.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
