Onasemnogene abeparvovec

The US FDA has approved a high dose regimen of Spinraza (nusinersen) for the treatment of spinal muscular atrophy, the agency and developer Biogen announced on March 30, 2026. Spinraza, an antisense oligonucleotide that modifies splicing of the SMN2 gene to increase production of functional SMN protein, was the first therapy approved for SMA when it received US FDA clearance in December 2016. The new regimen represents a dose optimization for a drug that has been administered intrathecally to SMA patients across all disease types for nearly a decade. The Muscular Dystrophy Association, which funded the early research by Adrian Krainer at Cold Spring Harbor Laboratory that underpinned the drug’s development, noted that the organization has invested more than USD 50 million in SMA research over its history. The high dose regimen delivers a higher concentration of nusinersen during both the loading and maintenance dosing phases compared to the original regimen. Spinraza is administered via intrathecal injection, requiring repeated lumbar punctures on a maintenance schedule of approximately every four months. The updated regimen was designed to increase drug exposure in the central nervous system while maintaining the safety profile established over the drug’s decade of clinical use, the company said. Higher CNS exposure may be particularly relevant in older or heavier patients, where drug distribution with standard dosing may be less optimal. The approval was based on findings from the DEVOTE study, a clinical trial evaluating the high dose regimen in SMA patients. The source material does not provide detailed efficacy endpoints, patient numbers, or comparative data from DEVOTE. Spinraza’s original approval rested on data from the Phase III ENDEAR trial in SMA Type 1, the Phase III CHERISH trial in SMA Types 2 and 3, and the NURTURE study in pre-symptomatic infants. Richard Finkel, an SMA investigator at St. Jude’s Children’s Research Hospital who participated in several of these trials, said the optimized dosing has “the opportunity to deliver even greater and more sustained benefits to patients across the spectrum of disease.” The approval arrives in a competitive treatment landscape for SMA that now includes three distinct therapeutic modalities. Novartis’s Zolgensma (onasemnogene abeparvovec), a one-time intravenous AAV9 gene therapy delivering a functional copy of the SMN1 gene, was approved in 2019 for patients under two years of age. Genentech/Roche’s Evrysdi (risdiplam), a daily oral small molecule SMN2 splicing modifier approved in 2020 for patients aged two months and older, offered the first non-invasive route of administration. Spinraza’s intrathecal delivery requirement has been a practical limitation, particularly for patients with scoliosis or prior spinal fusion, and the drug has faced competitive pressure from these alternatives. The dose optimization may represent Biogen’s effort to strengthen the clinical rationale for continued or new use of Spinraza in a field where treatment decisions increasingly weigh efficacy, route of administration, and long-term durability. Despite the availability of three approved therapies, incomplete motor recovery remains common, particularly among patients treated after symptom onset, and questions persist around optimal sequencing or combination of available treatments. No combination regimen has received regulatory approval as of March 2026. The expansion of newborn screening programs for SMA across the United States and internationally has shifted treatment increasingly toward pre-symptomatic initiation, where outcomes across all three therapies tend to be most favorable. [Word count: 498] Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Roche, citing discouraging clinical trial results, has chosen to stop developing an experimental antibody drug as a potential treatment for two rare, muscle-wasting diseases.Known as emugrobart, the drug is meant to lower levels of a protein, myostatin, that both breaks down muscle fibers and restricts new muscle from building. Chugai Pharmaceutical, a Japan-based Roche subsidiary, designed it to bind to, and thereby keep inactive, a precursor protein to myostatin. Researchers have been testing emugrobart against spinal muscular atrophy and facioscapulohumeral muscular dystrophy, and as a way to retain lean muscle mass in people with obesity who are taking a GLP-1 medication.In SMA, Roche has been sponsoring a two-part trial to evaluate whether its worth adding emugrobart on top of the companys already approved therapy Evrysdi. The experiment aimed to recruit around 250 patients, and focused on safety as well as biological markers of muscle health.But, after a comprehensive assessment of all the evidence gathered in the first part of the experiment, the Swiss pharmaceutical giant determined its drug did not consistently deliver enough improvements in muscle growth and motor function to warrant further study.We recognise this news will be disappointing to the SMA community, wrote Louisa Townson, Roches patient partnership director for rare diseases, in a Thursday letter to a European patient advocacy group, explaining the companys decision.Townson added that Roche remains committed to advancing quality care for people living with SMA, and plans to share data from the trial at an upcoming medical conference so that this research can also help to inform the development of future treatments. She penned a similar letter to the facioscapulohumeral muscular dystrophy community, referring to a separate mid-stage trial that also generated less-than-ideal results.While estimates vary, the number of people living with SMA in the U.S. is generally thought to be in the range of 10,000 to around 30,000. It mostly affects young children, and is caused by alterations in a gene responsible for making survival motor neuron, a protein crucial for signaling between muscles and the nervous system.While historically fatal, the prognosis for this disease changed drastically over the past decade as three medicines Biogens antisense drug Spinraza, Novartis gene therapy Zolgensma and Evrysdi became available to patients. Each of these works by boosting survival motor neuron levels.Last year, sales of Evrysdi, which is taken orally, totaled 1.76 billion Swiss francs, or roughly $2.1 billion. Roche had been hoping to expand the business with emugrobart, which, by targeting myostatin, offered a different route to addressing SMA. Its a strategy adopted by other developers as well, including Biohaven and Massachusetts-based Scholar Rock.Eric Schmidt, an analyst at Cantor Fitzgerald who covers Scholar Rock, highlighted how its drug apitegromab has been ahead of Roches for several years, and is now being developed as both an under-the-skin shot along with a monthly intravenous infusion. Roche, therefore, offered little in the way of differentiation, according to Schmidt.We now expect apitegromab to have near complete market share as the lone muscle-building therapy for SMA patients, the analyst wrote in a Thursday note to clients, adding that his team sees an approval from the Food and Drug Administration this year as a “when not if“ event. The Cantor team expects the therapy, which the FDA rejected last year due to manufacturing concerns,to have multibillion-dollar potential.Roches update is a clear win for Scholar Rock ahead of a likely FDA approval, wrote Raymond James analyst Martin Auster in his own note to clients.Shares of Scholar Rock were up 1.5%, to a little over $46 apiece, in late morning trading Friday. Biohaven shares were up 2.5%. '