FDA fast tracks Dyscorban for treating heart problems in Duchenne
Trial data show therapy lowers heart damage markers in people with DMD
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The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyscorban (ifetroban), Cumberland Pharmaceuticals’ treatment candidate for heart problems in Duchenne muscular dystrophy (DMD).
Dyscorban has been tested in the Phase 2 FIGHT DMD trial (NCT03340675), with data showing that it improved heart function and reduced markers of heart damage in individuals with DMD.
Fast track status is designed to expedite the development of therapies for serious conditions with a significant unmet medical need. It provides developers with more frequent interactions with the FDA, enabling a company to submit portions of a new drug application as they are ready instead of having to wait for the whole package to be completed, as is normally the process. The status award may also make a treatment eligible for priority review.
“This designation, combined with our breakthrough Phase 2 results, positions us to work closely with the FDA through more frequent interactions and expedited review processes to advance this promising heart-targeted therapy for DMD patients as efficiently as possible,” A.J. Kazimi, Cumberland’s founder and CEO, said in a company press release.
Duchenne is caused by mutations in the DMD gene that result in a lack of functional dystrophin, a protein that helps protect muscles from injury. The condition leads to progressive muscle wasting and can cause damage to the heart muscles, which pump blood throughout the body. There are approved therapies, however, that target heart disease in DMD.
Dyscorban is a once-daily oral medication designed for cardiomyopathy — a general term for diseases of the heart muscle — associated with Duchenne. It works by blocking the thromboxane-prostanoid receptor to reduce fibrosis, or scarring, and preserve heart function.
In trial, Dyscorban improved heart function in DMD
The FIGHT DMD trial enrolled 46 males, ages 7 and older, who were randomly assigned to receive one of two doses of Dyscorban (150 mg or 300 mg per day), or a placebo, for one year.
Results from the trial showed that the higher dose was associated with a 3.3% improvement in left ventricular ejection fraction (LVEF) — an indicator of how well the heart pumps blood with each heartbeat — compared with the 1.5% seen in those given the placebo. Compared with both those on the placebo and an external group of matched, untreated DMD patients, the relative LVEF improvement with Dyscorban amounted to 5.4%.
Treatment also led to reduced blood levels of cardiac damage markers, specifically, NT-proBNP and cardiac troponin I. In contrast, those levels were increased in the placebo group.
Dyscorban therapy was well tolerated, the developer noted.
Participants who completed the main trial opted to continue in an open-label extension, in which they are receiving high-dose Dyscorban for up to three years.
The FIGHT DMD trial was funded by a $1 million FDA grant, part of the agency’s orphan products grants program.
The experimental therapy has also been granted orphan drug and rare pediatric disease designation by the FDA. Orphan drug status is awarded to therapies for rare diseases, those affecting fewer than 200,000 people in the U.S., and comes with incentives for the developer. Rare pediatric disease designation is given to medications for conditions affecting children.
