Risdiplam (RG7916) is a drug that is produced to treat the spinal muscular atrophy (SMA). PTC Therapeutics, Inc. announced that priority review has been granted by the U.S. FDA for the approval of new drug risdiplam. FDA approves Prescription Drug User Fee Act (PDUFA) on May 24, 2020.
PTC, a science-driven and global biopharmaceutical company, is involved in the discovery, development and commercial sale of the drugs for treating the rare diseases after the clinical trial approvals. The main goal of the PTC is to introduce the new drugs at the commercial level that enhance that investment in a robust pipeline of transformative medicine. Also, it provides better access to the patients for the cure of such type or rare disorders that usually lack the medical needs.
SMA is a genetic disorder of neuro muscles. It is the leading cause of the high mortality rate in infants. It is due to a mutation in the gene (SMN1) of the survival of motor neurons that resulted in the low production of SMN proteins.
The inadequate amount of this protein leads to the loss of motor neurons progressively. It will result in muscular atrophy and lead to death in many cases i.e. 1 in 11,000 newborn kids.
According to Stuart W. Peltz, Ph.D. the chief executive officer of PTC therapeutics it the crucial step that FDA accepts the NDA making the drug risdiplam for treating the patients round the U.S. it is the first oral molecule targeting splicing that produced from the proprietary splicing platform.
The approval of risdiplam’s NDA is the result of SAM population by administrating the drugs with patients type 1, type 2 and type 3. The results show improvements in the motor function of the patients and developmental milestones. The oral administration of the drug in the SMA patients will be efficient in treating the disease.
The priority review granted by FDA approved the drug if it would fulfill the following terms and conditions i.e. safety level, effective for the treatment and diagnosis, preventing the adverse effects by comparing with a standard application.
Risdiplam is a drug that is on the investigation stage. it is studied on a broader level of patients who suffer from SMA. The drug is administrated in the patients from time f birth to 60 years. The drug function is evaluated by measuring the level of SMA proteins in patients. It causes the SMA 2 gene to produced more functional proteins, that in normal condition produce a low amount of protein.
Two studies involved in the NDA filing, that are based on the12 month data from theses these studies FIREFISH and SUNFISH on the dose-finding portion of the drug. Also, from the clinical, preclinical, pharmacodynamic and pharmacokinetic data. A double-blinded study SUNFISH, that contains two-part, one is the placebo-controlled trails in the patients with type 3 or 2 SMA from the age 2 years to 25 years.
FIREFISH, an open-label study, it is a two-part study with clinical trials of risdiplam in the SMA infants’ patients. Both the studies and their results will be presented at the medical congress. The SMA program is working by the collaboration of PTC with the SMA Foundation and Roche.