FDA Approves Third Duchenne Muscular Dystrophy Treatment in Nine Months

2024-03-22

临床3期临床结果上市批准基因疗法

Pictured: A boy sits in a wheelchair on a beach/iStock, ChiccoDodiFC A young biotech has its first approved product after the FDA greenlit ITF Therapeutics’ Duvyzat (givinostat) Thursday as the third new treatment for Duchenne muscular dystrophy since last June. The approval of Duvyzat for patients six years and older was granted to ITF’s parent company, Italfarmaco. Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of Duchenne muscular dystrophy (DMD), according to the FDA’s announcement. Duvyzat “adds to list of approved treatments for families facing this devastating disease and is an important step forward in accelerating transformative treatments for everyone independent of their genetic mutation,” Debra Miller, founder and CEO of CureDuchenne, said in a statement emailed to BioSpace. Leading up to the decision, Italfarmaco reported final results from the Phase III Epidys trial, which met the primary endpoint of a change in the four-stair climb assessment from baseline to 72 weeks. Duvyzat also showed “favorable outcomes” in key secondary measures, according to the company, including on the North Star Ambulatory Assessment (NSAA), a 17-item scale that measures motor function skills. On the NSAA, patients treated with the new drug saw less worsening compared to placebo after 18 months. Patients received a standard-of-care steroid regimen throughout the trial. Duvyzat was generally well tolerated, with no treatment-related severe or serious adverse events. Craig McDonald, chair of the Department of Physical Medicine & Rehabilitation at UC Davis Health and the study’s lead U.S. investigator, told BioSpace he has been “quite impressed” with the data on Duvyzat. McDonald, who has treated nine patients with the drug, said he has “a number of patients” who are still ambulatory at 16 or 17 years old. DMD patients on steroid treatment typically lose the ability to walk at around 10 to 13 years of age, McDonald noted. Duvyzat is a small molecule that acts by inhibiting histone deacetylases (HDACs), a group of enzymes that modulate gene and protein expression in the muscle. “Deregulation of HDACs is a major consequence of the lack of dystrophin associated with DMD,” Matt Trudeau, head of ITF, told BioSpace in an interview ahead of the approval. Third New DMD Treatment in Nine Months While ITF only incorporated in January 2024, Duvyzat has been in development at Italfarmaco for the past six to eight years, Trudeau said. He added that ITF has the right team in place to get the drug to patients quickly. “We’re bringing in a team that knows how to turn systems on quickly and move with certain purpose and urgency and passion for the patients that we hope to serve.” The new company has leveraged the “rare disease talent epicenter” of Boston and Cambridge, Mass., to build its team, which comprises former employees of Biogen, bluebird bio and Genzyme, companies that have proven success in the rare disease space, Trudeau noted. While Trudeau said ITF’s “express purpose” is to create the systems, processes and communication tools to bring Duvyzat to DMD patients, the opportunity exists to establish a rare disease business in the U.S. For the moment, though, he said ITF is “laser-focused on DMD.” They’re not the only ones paying attention to the degenerative disease. In June 2023, the FDA greenlit Sarepta’s Elevidys as the first gene therapy for DMD. Then, in October, Santhera Pharmaceuticals got the nod for Agamree, a novel corticosteroid that will be marketed in the U.S. by Catalyst Pharmaceuticals. The DMD pipeline is also flush with new candidates, including Pfizer’s mini-dystrophin gene therapy fordadistrogene movaparvovec, for which Phase III data are anticipated in the second half of 2024, and REGENXBIO’s one-time gene therapy. REGENXBIO reported positive efficacy data earlier this month from the first patient to receive a higher dose level of the treatment in a Phase I/II trial. “I think we’ve seen some exciting therapeutics come along under investigation in recent years,” McDonald said. Heather McKenzie is a senior editor at BioSpace. You can reach her at heather.mckenzie@biospace.com. Also follow her on LinkedIn.