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Givinostat from Italfarmaco gets FDA rare pediatric disease designation in duchenne muscular dystrophy

Announces completed enrollment in EPIDYS Phase 3 trial

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The Italfarmaco Group provided an update on the development of Givinostat, its proprietary histone deacetylase (HDAC) inhibitor, in boys with Duchenne Muscular Dystrophy (DMD). The US Food and Drug Administration (FDA) granted a Rare Pediatric Disease designation to Givinostat for the treatment of DMD, which allows an expedited review process for new treatment modalities. The company also announced the completion of patient enrollment in the EPIDYS Phase 3 trial on September 25th, 2020 and remains on track with its proposed timelines for reporting topline data in June 2022.

“This is the third regulatory designation we have received from the FDA for Givinostat after the Orphan Drug designation and Fast Track designation and reflects the agency’s recognition of Givinostat’s potential to treat DMD, a devastating genetic disease,” said Paolo Bettica, Chief Medical Officer at the Italfarmaco Group.

The Rare Pediatric Disease designation is granted by the FDA to encourage the development of new drugs for serious or life-threatening manifestations of a disease or condition that primarily affect children 18 years of age and younger. Subject to FDA approval of Givinostat for the treatment of DMD, Italfarmaco would be eligible to receive a voucher, which may be redeemed to receive priority review for a subsequent marketing application for a different product candidate or which could be sold or transferred.

In April 2020, Italfarmaco announced that interim data from the EPIDYS Phase 3 clinical trial was reviewed by an Independent Data Monitoring Committee and that the trial was recommended to continue based on the lower limb muscle fat infiltration seen in the Magnetic Resonance Spectroscopy (MRS) images of DMD patients with Givinostat treatment compared to the placebo group after 12 months of treatment. These MRS results corroborate the histological results of the company’s Phase 2 study, which revealed slower disease progression in boys treated with Givinostat and on a stable regimen of steroids. The Independent Data Monitoring Committee, which regularly monitors and reviews the study, confirmed the safety of the treatment with Givinostat. With trial enrollment completed, the last patient is expected to complete the 72-week treatment period in the first quarter of 2022. Italfarmaco is expected to report topline data from the Phase 3 EPIDYS trial in June 2022.

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