According to a story from BioSpace, the pharmaceutical company Scholar Rock recently announced that its drug candidate SRK-015 was recently granted Orphan Drug Designation by the U.S. FDA. The drug is in development for the treatment of spinal muscular atrophy. The company claims that SRK-015 has the potential to prevent or even reverse the muscle degeneration that occurs in the disease, and is designed to be used alone or in combination with other currently available treatments.
Spinal muscular atrophy (SMA) is a rare disorder in which the motor neurons that are responsible for muscle movement start to die off, and as a result, the wasting of the skeletal muscles occur. This is caused by a genetic mutation that results in the body not producing a normal amount of SMN, a protein that is expressed by all cells and is essential for the survival of motor neurons. The breathing muscles and the muscles of the upper arms and legs are often the first that are affected. Spinal muscular atrophy varies significantly in its severity. In its most severe forms, patients may die within weeks after birth, but late onset variants often do not have an effect on lifespan. To learn more about spinal muscular atrophy, click here.
The announcement came earlier than the company anticipated, but it is not a complete surprise since there are few drugs currently available for the treatment of SMA. Nusinersen is currently the only available medication designed specifically for this disorder. Orphan drug designation is typically only given to clinical stage drugs that demonstrate a significant advance in treatment outcomes, address a medical need that is not being met by currently available medications, and treats a disease that is considered rare (affecting less than 200,000 in the country). The designation is meant to provide incentives for companies to develop treatments for rare diseases, and companies get exempted from paying certain fees and a seven year window of market exclusivity if the drug gets approved.
