FDA Verdict Awaited: ITF Enters Growing DMD Treatment Arena

The ongoing annual conference of the Muscular Dystrophy Association is a significant event in the medical field, especially with the recent advancements in the treatment of Duchenne muscular dystrophy (DMD). Sarepta Therapeutics has reported a high demand for its gene therapy, Elevidys, and Catalyst Pharmaceuticals is looking forward to the launch of Agamree in the first quarter of 2024. Other companies, including ITF Therapeutics, are also showing interest in the DMD treatment market.

ITF, the U.S. branch of the Italian pharmaceutical group Italfarmaco, is preparing for an FDA PDUFA date on March 21 for its drug candidate, givinostat, which is designed to treat DMD. The company initially anticipated a decision by the end of 2023, but the FDA extended the review period by three months. Givinostat, a small molecule that inhibits histone deacetylases (HDACs), has shown promising results in Phase III trials, potentially reducing the decline in mobility and muscle function in DMD patients.

The drug's primary endpoint was achieved in a trial involving 179 ambulant boys with DMD, aged six and above. The trial compared the effects of givinostat combined with standard care—steroids—and a placebo group that received steroids only. HDACs are enzymes that regulate gene and protein expression in muscles, and their deregulation is linked to the lack of dystrophin in DMD. Givinostat is believed to inhibit the overactivity of HDACs, which could help slow down muscle damage and deterioration.

Dr. Craig McDonald, the lead U.S. investigator for givinostat's Phase III trial, has noted the drug's positive impact on DNA methylation and gene expression in muscle tissue. He has treated nine patients with givinostat and is impressed with their progress.

The DMD treatment landscape is evolving, with the FDA's approval of Sarepta's Elevidys as the first gene therapy for DMD. Despite missing its primary endpoint in a randomized trial, it was approved under the FDA's accelerated pathway. However, the therapy's effectiveness is still under debate, with some experts noting that it falls short of expectations.

Steroid hormones, such as Agamree, can be used alongside gene therapies to mitigate inflammatory side effects but come with their own set of side effects. Agamree, recently approved by the FDA, is an oral suspension steroid indicated for DMD patients aged two and older. Catalyst Pharmaceuticals plans to launch the drug by the end of the month.

The DMD pipeline is rich and diverse, with various companies expecting to release data this year. Gene therapies, exon-skipping approaches, and other modalities are in development, offering hope for more effective treatments in the future. The approval of any new therapy is seen as a positive step forward for DMD patients, as it expands the range of available treatment options.