Risdiplam

FDA clears high-dose Spinraza on second go-roundBoost of $500M for J&J autoimmune drugBMS's Camzyos readout in teens with obstructive HCMBoehringer licenses stroke antibody therapy from EmfretFDA clears high-dose Spinraza on second go-roundThe FDA on Monday approved a high-dose formulation of Spinraza (nusinersen) to treat spinal muscular atrophy (SMA), a win for Biogen after the US regulator had earlier rejected an application for the antisense oligonucleotide (ASO). The September complete response letter did not flag any issues with the supporting efficacy and safety data, but the FDA had requested a technical information update be included in the chemistry, manufacturing and controls (CMC) portion of the drug's application.Biogen said the high-dose regimen — which involves two 50-mg loading doses given two weeks apart, followed by 28-mg maintenance doses every four months — will be available in the US "in the coming weeks." The approval was based on findings from the Phase II/III DEVOTE study in treatment-naïve, symptomatic infants with SMA, in which high-dose Spinraza met its primary endpoint of significantly improving motor function compared to untreated matched controls.With increasing competition from Roche's oral small molecule Evrysdi (risdiplam) in the SMA space, Biogen has been hoping the new Spinraza formulation will refresh interest in the ASO. While its intrathecal delivery mechanism puts it at a dosing disadvantage against oral Evrysdi, Biogen President and CEO Christopher Viehbacher previously emphasised that when it comes to SMA treatments, "It's efficacy that matters, and that's really the story of Spinraza. The high dose, I think, will be important because you can get to the therapeutic levels of drug that you need much faster" (see – Spotlight On: How will newcomers reshape the SMA market?).-Elizabeth EatonBoost of $500M for J&J autoimmune drugJohnson & Johnson and Royalty Pharma have launched a $500-million collaboration aimed at advancing JNJ-4804, a co-antibody therapy that blocks the complementary IL-23 and TNF pathways. The joint funding agreement covers 2026 and 2027.JNJ-4804 is currently in Phase II for ulcerative colitis, Crohn's disease and psoriatic arthritis, according to J&J's website. A few years ago, as the company prepared for Stelara (ustekinumab) generics to hit the market, it highlighted JNJ-4804 as one of over a dozen assets that could potentially generate between $1 billion and $5 billion in peak sales."Royalty Pharma has a strong track record of investing in immunology, beginning with early disease-modifying biologics including TNF inhibitors," noted Royalty CEO Pablo Legorreta in a  release Monday. "We believe JNJ-4804 holds substantial promise for patients with chronic immune-mediated diseases."-Anna BratulicBMS's Camzyos readout in teens with obstructive HCMAfter top-lining upbeat results in January, Bristol Myers Squibb fleshed out detailed late-stage data for Camzyos (mavacamten) in adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), with the aim of seeking a broader label for the cardiac myosin inhibitor (CMI) in this age group. Findings from the Phase III SCOUT-HCM trial — presented at the American College of Cardiology (ACC) meeting — showed that at week 28, Camzyos delivered a significant and clinically meaningful reduction from baseline in Valsalva left ventricular outflow tract (LVOT) gradient versus placebo, with a least-squares mean difference (LS MD) of -48.0 mmHg. "The SCOUT-HCM results underscore the potential for Camzyos to become the first CMI for adolescents," said Cristian Massacesi, chief medical officer at BMS. "With these meaningful safety and efficacy data, we are excited about the potential to provide a paradigm-changing treatment."Camzyos was first approved by the FDA in 2022 to treat oHCM in adults, and secured a revised label last year that cut down certain monitoring requirements. -Pavan KamatBoehringer licenses stroke antibody therapy from EmfretBoehringer Ingelheim on Monday licensed a preclinical antibody-based asset from Emfret Analytics in development for acute ischemic stroke. While the partners didn't disclose specific details of the deal, Emfret will receive an upfront payment and is eligible for development, regulatory and commercial milestones, plus tiered royalties.The candidate targets the platelet collagen/fibrin receptor glycoprotein VI (GPVI) and is designed to inhibit the biological pathway — which is a driver of harmful thrombo-inflammatory responses in acute ischemic stroke — whilst maintaining normal hemostasis. The antibody programme, intended to be given on top of standard-of-care reperfusion therapies, has shown strong protection from infarct growth in preclinical models."By addressing a central thrombo-inflammatory target, this collaboration explores a novel complementary therapeutic approach with the potential to further improve outcomes when added to established therapies," said Bernhard Nieswandt, Emfret's cofounder and chief scientist.-Elizabeth Eaton

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