The FDA is slated to decide on approving Capricor’s drug for the treatment of Duchenne muscular dystrophy by November 22. HomeNewsMarketsEquityCAPR Stock Rises As Clinical Trial Investigators Write To FDA Urging Approval For Deramiocel · Advertisement|Remove ads. The FDA is slated to decide on approving Capricor’s drug for the treatment of Duchenne muscular dystrophy by November 22. In this photo illustration, the lCapricor Therapeutics logo is seen displayed on the screen of a tablet. No approved therapy has shown efficacy in this population, the investigators noted. They pointed to preserved upper-limb function in the trial, or the ability to self-feed, bring a hand to the mouth, or operate a powered wheelchair or communication device, as meaningful improvement. The group also referenced the FDA’s Rare Disease Framework Initiative, which encourages flexibility and consistency when reviewing therapies for small patient populations, and noted that all the physicians involved in the trial believe it would be “unethical” to proceed with another trial to prove efficacy, given existing data. Shares of Capricor Therapeutics (CAPR) climbed roughly 14% on Tuesday as a public letter from clinical investigators added momentum ahead of a pivotal regulatory decision on the company’s therapy Deramiocel. The FDA is slated to decide on approving the drug for the treatment of Duchenne muscular dystrophy by November 22. Duchenne muscular dystrophy (DMD) is a rare, inherited genetic disorder that causes progressive muscle degeneration and weakness that presents predominantly in young boys. The company resubmitted the application earlier this year with late-stage HOPE-3 trial data, and the agency said it will decide on the application by August 22. However, an advisory committee met at the end of July and voted 9–3 that the evidence did not support effectiveness for cardiomyopathy. Advertisement|Remove ads. Capricor then submitted additional 24-month clinical data analyses from the HOPE-3 trial and asked FDA to consider a refined label built on the trial’s primary endpoint—preservation of upper-limb function—rather than cardiomyopathy.

This week’s news roundup covers the upcoming Drug Discovery Innovators & Investors Conference in London, as well as new initiatives to accelerate rare disease drug development, promising preclinical data for Duchenne muscular dystrophy, clinical trial progress in advanced Parkinson’s disease, ... This week’s news roundup covers the upcoming Drug Discovery Innovators & Investors Conference in London, as well as new initiatives to accelerate rare disease drug development, promising preclinical data for Duchenne muscular dystrophy, clinical trial progress in advanced Parkinson’s disease, and a potential new drug target for breast cancer. The DDW Fall 2026 issue examines precision therapies for Parkinson's Disease, what the resurgence of neuroscience means for drug discovery and why cancer vaccines depend on DNA synthesis. Other features in the Fall issue include: From investment to innovation - why we've launched a new event series The three generations of obesity medications Rethinking paediatric oncology treatments Read More ScionxBio, a preclinical-stage biotechnology company developing next-generation mRNA therapeutics, has entered into a worldwide strategic collaboration and licensing agreement with Sanofi to develop and commercialise extended exposure versions of certain Sanofi mRNA candidates by leveraging ScionxBio’s AI-driven platform. Cancer Research Immunology / Virology & Vaccines Advanced Therapies Neuroscience Multiomics Artificial Intelligence Automation & Robotics Therapeutic Antibodies Microbiome Drug Discovery Cell Science

A phase 1/2 trial of delpacibart zotadirsen shows promising safety and efficacy for treating Duchenne muscular dystrophy mutations that are amenable to exon 44 skipping. This randomized, placebo-controlled study provides critical insights into potential therapeutic options for this rare disease.

Solid Biosciences Inc is a ... with rare diseases. The company’s research platform is centered on adeno-associated virus (AAV) gene therapy and other genetic medicine technologies intended to address the underlying causes of disease. Solid Biosciences’ lead program is SGT-003, an investigational gene therapy being developed for Duchenne muscular dystrophy, a severe inherited neuromuscular disorder... Solid Biosciences Inc is a biotechnology company focused on developing genetic medicines for patients with rare diseases. The company’s research platform is centered on adeno-associated virus (AAV) gene therapy and other genetic medicine technologies intended to address the underlying causes of disease. Solid Biosciences’ lead program is SGT-003, an investigational gene therapy being developed for Duchenne muscular dystrophy, a severe inherited neuromuscular disorder. Solid Biosciences Inc. (NASDAQ:SLDB – Get Free Report) has received a consensus rating of “Moderate Buy” from the thirteen analysts that are covering the company, Marketbeat Ratings reports. One research analyst has rated the stock with a sell recommendation and twelve have issued a buy recommendation on the company. Celldex Therapeutics, Inc.

Jefferies analysts made the prediction after Sarepta Therapeutics, which has endured nothing short of a saga with the Duchenne muscular dystrophy gene therapy Elevidys, presented data this week showing its clinically meaningful benefit in older patients who are able to walk.