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FDA clears first-of-its-kind Duchenne drug for broad use

Dive Brief:

  • The Food and Drug Administration on Thursday approved a first-of-its-kind drug for Duchenne muscular dystrophy, clearing Duvyzat, a medicine developed by Italian pharmaceutical company Italfarmaco and to be sold in the U.S. by its subsidiary ITF Therapeutics.

  • Duvyzat is the first non-steroidal treatment approved for use in all Duchenne patients who are at least six years of age, regardless of their disease’s genetic underpinnings. The drug is a pill designed to slow inflammation and muscle loss. In testing, it was associated with statistically significant benefits, compared to a placebo, on measures of motor function.

  • The drug’s price hasn’t been determined yet, according to an ITF spokesperson. Its labeling information requires healthcare providers to evaluate patients’ platelet counts and triglycerides before prescribing Duvyzat, and to monitor levels of each afterwards. The most common side effects associated with treatment were diarrhea, stomach pain, nausea and vomiting.

Dive Insight:

The treatment landscape for Duchenne, a progressive and typically fatal neuromuscular disease, has changed significantly in recent years. Duvyzat is the latest example.

Since 2016, the FDA has approved four “exon skippers,” which are thought to modestly delay disease progression. Last year the agency also greenlit the first gene therapy for the condition, Sarepta Therapeutics’ Elevidys, which patients and researchers hope can provide more substantive benefits. The FDA cleared a newer type of steroid drug from Santhera Pharmaceuticals, Agamree, in 2023 as well.

Yet nearly all of those drugs are for small subsets of Duchenne patients. Exon skippers, for instance, are each tailored to people with specific genetic mutations. Elevidys is only cleared for use in young children who can still walk and don’t have genetic mutations that might blunt the treatment’s effects or raise its safety risks. Sarepta aims to expand Elevidys’ use, but there are questions as to what type of label the FDA will be open to given the therapy’s mixed results in testing.

The most broadly used therapies are steroids. While they can slow the disease, they aren’t curative. The approval of Duvyzat now gives patients a way to potentially boost their effects.

The drug is a so-called HDAC inhibitor, a type of enzyme-targeting medicine best known for its use treating cancer. Duvyzat works by blocking HDAC overactivity that leads to muscle damage, in turn slowing the breakdown of muscle fibers tied to the disease. When given alongside steroids, the drug led to benefits on a patient’s ability to climb stairs, as well as on a functional scale that measures movement. The findings were recently published in The Lancet.

The drug’s approval is “an important step forward in accelerating transformative treatments for everyone independent of their genetic mutation,” said Debra Miller, the founder and CEO of patient advocacy group CureDuchenne, in an emailed statement.

In a research note last November, RBC Capital Markets analyst Brian Abrahams wrote that there is a desire among patients “to move towards combination therapy” and “develop a universal treatment protocol for patients.” Patient advocates had specifically pointed to Duvyzat as therapies the community was “most excited for,” he wrote.

In an email to BioPharma Dive, an ITF spokesperson said the company is “committed to making the drug available to appropriate patients with [Duchenne] in the U.S. as quickly as possible.”