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Italfarmaco nabs FDA approval for new Duchenne med, teeing up sparring match with Sarepta

2024-03-22

上市批准临床3期临床结果

With an approval in hand, Italfarmaco’s next goal is to get Duvyzat to U.S. patients “as quickly as possible,” according to the company’s chief medical officer Paolo Bettica, M.D., Ph.D.

At a watershed moment for Italfarmacoscular dystrophy (DMD) Duvyzatnt, privately held Italian drugmaker Italfarmaco has added a new therapeutic option to the armamentarium against the debilitating muscle-wasting disorder.

Late Thursday, the U.S. FDDuchenne muscular dystrophy (DMD)’s histone deacetylase (HDAC) inhibitor DuvyzItalfarmacoown as givinostat.muscle-wasting disorder

The approval makes the pFDAuct the first nonsterItalfarmacoapphistone deacetylase (HDAC) inhibitoreDuvyzatiants of DMD in givinostatthe FDA said in an approval announcement. Duvyzat, which is taken orally, is specifically cleared to treat DMD in patients ages 6 and older, Italfarmaco said in a separate release.

DMD, which is the most common form of muscular dystrophy around the world, is a severe neuromuscular genetic diseasDMDarked by progressiFDAmuscle weakness and degeneration. TDuvyzatase is caused by mutations in the dystrophin gene that leaDMDo an absence of functional dystrophin proteins, in turn leaving muscle fibers prone to injury, inflammation and impaired regeneration and replacement. DMD primarily affects boys, but girls can be carriers of the disease, according to the Muscular Dystrophy Association.

DMDsting a unique mechanism of action,muscular dystrophymodulating the deregulated actineuromuscular genetic diseaseic muscle, progressive muscle weakness major consequence of the dystrophin deficit associated with dystrophinhibiting HDAC overactivity, Duvyzat is desigdystrophin proteinscascade of events leading to muscle damage, ultiminflammationg down muscle deterioration, Italfarmaco saiDMD Muscular Dystrophy

Citing the “tremendous unmet need” for Duvyzatrapies that can help a broad range of patients, CHDACsMcDonald, M.D., an investigator on Duvyzat’s pivotal phase 3 trial, said in a stdystrophinat Italfarmaco’s drug hasDMDe potential to bHDACe a “key componDuvyzatthe standard of care for people living with DMD.”

The FDA based its approval on results fDMD Italfarmaco’s phase 3 EPIDYS trial, which looked at 179 boys ages 6 and up who received eithDuvyzatzat twice daily or placebo in addition to glucocorticosteroid treatment. At the trial’s 18-month mark, patients on Duvyzat showed significantly less decline in thDMDime it took to complete a four-stair climb measure of muscle function.

WithFDA approval in hand, Italfarmaco’s nexItalfarmacoo get Duvyzat to U.S. patients “as quickly as possible,” according to the company’s Duvyzatedical officer Paolo Bettica, M.D., Ph.D.Duvyzat

To that end, the company hItalfarmacocantly expanded its U.SDuvyzatnce” with the launch of subsidiary ITF Therapeutics, which will handle marketing for the drug in the States. Italfarmaco said Thursday that ITF is working closely with healthcare providers, patient advocacy groups and payers to help get Duvyzat on pharmacy shelves.

Privately held Italfarmaco was established in 1938 and currently operates in Italy and abroad from itsITF Therapeutics Milan. The company mainly makes drugs for cardiovascular,Italfarmacoologic and gynecological disorders, according to its website.

With its DuvyzaItalfarmacoht, Italfarmaco will now have to go up against the likes of Sarepta Therapeutics, which boasts a suite of exon-skipping therapies for DMD such as Exondys 51, Vyondys and Amgynecological disorders

MeanwhileDuvyzatta made a splaItalfarmacomer with the FDA approval of its DMD gene theSarepta Therapeutics has become one of the world’s most expensive drugs wiDMDa price tag of $3.2 million.

Elevidys works by delivering a micro-dystrophin-encodiFDAgene into a patieDMDs muscle tissue to prompt production of the micro-dystrophin protein.

As Sarepta waits on a potential label expansion for Elevidys, the company said in late February that it’s seen early succmicro-dystrophin proteiny’s launch. The drug brought home total sales of $200.4 million last year, with $131.3 million coming in 2023’s fourth quarter.

ElsSarepta Santhera Pharmaceuticals and Catalyst Pharmaceuticals in October received an FDA nod for their own DMD therapy—a dissociative steroid known as Agamree, or vamorolone.

And, just tSanthera Pharmaceuticalscs reCatalyst Pharmaceuticals its application for theFDAnsense mutation DMDDMDndidate Tradissociative steroidy the middAgamreehe yevamoroloneve comes after the U.S. regulator snubbed PTC’s drug in 2016 and 2017.