Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” grants program. Recently, a clinical trial investigating vamorolone as a treatment for Becker muscular dystrophy received $1.2 million from the FDA under this program.
This dissociative steroid drug has shown prior success with Duchenne muscular dystrophy (DMD), leading medical professionals to believe it could have similar results in Becker muscular dystrophy patients. It binds to the same receptor that corticosteroids do; however, it has a different downstream activity.
As corticosteroids can present a number of side effects and safety concerns, patients and medical professionals alike are very excited about the possibility of vamorolone as a new standard of care. If it is able to provide positive results, it could greatly improve the lives of patients.
It has already received the Orphan Drug designation in both the U.S. and Europe for DMD, alongside the Rare Pediatric Disease and Fast Track designations solely from the U.S. Its indications don’t stop there; the U.K.’s MHRA has also granted Promising Innovative Medicine (PIM) status for DMD.
Grants for Vamorolone
The $1.2 million from the FDA adds to grants that this drug candidate has already received. NIH, the Foundation to Eradicate Duchenne, and NIAMS have already dedicated money to this cause. This money will go towards a clinical trial that evaluates vamorolone as a treatment for Becker muscular dystrophy in children and adults.
The study will last for 24 weeks, and it follows the highly successful VISION-DMD trial.
About Becker Muscular Dystrophy
Becker muscular dystrophy is one of the many forms of muscular dystrophy. This type is characterized by progressive weakness and loss of muscle mass in the cardiac and skeletal muscles. It typically impacts males, with onset occurring between ages five and fifteen. Weakness tends to begin in the pelvis and legs, resulting in symptoms like falling, difficulty walking, and problems with muscle skills. As the heart muscles weaken, cardiomyopathy becomes another symptom. Other effects are fatigue, cognitive problems, scoliosis, difficulty breathing, contractures, a loss of balance and coordination, and muscle weakness in various regions throughout the body. All of these symptoms are the result of a mutation in the DMD gene, which is inherited in an X-linked recessive pattern. There is currently no cure, and treatment is symptomatic.
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