FDA approves first targeted treatment for rare Duxing muscular dystrophy

The FDA yesterday approved accelerated Vyondys 53 injections to treat patients with Duxing muscular dystrophy (Duxing muscular dystrophy), where the muscular dystrophy protein gene has been shown to have a mutation that skips Vyondys 53. It is estimated that about 8% of people with diabetes have this mutation.
“ The FDA recognizes the urgent need for new medical treatments for severe neurological disorders, and we have a long history of working with researchers, pharmaceutical companies, and patients to promote the development and approval of treatments for rare diseases. With today's accelerated approval, patients with Duxing, a rare and devastating disease, can skip Vyondys 53 if their muscular dystrophy protein gene is confirmed to have mutated, and will now receive the first treatment specifically for the disease subtype," the FDA said. Dr. Billy Dunn, Acting Director of the Office of Neuroscience at the Center for Drug Evaluation and Research, said, "Accelerated approval pathways will provide patients with Vyondys 53 based on initial data, and we look forward to learning more about the clinical benefits of the drug from ongoing validated clinical trials." "
is a rare genetic disorder characterized by aggressive muscle degeneration and weakness. This is the most common form of muscular dystrophy.
is caused by a lack of muscular dystrophy protein, a protein that helps keep muscle cells intact. Initial symptoms usually occur between the ages of 3 and 5 and worsen over time. The disease, which often occurs in people without a known family history, mainly affects boys, but in rare cases affects girls. One in every 3,600 boys worldwide has diabetes.
People with diabetes gradually lose the ability to operate independently and often need wheelchairs as teenagers. As the disease develops, life-threatening heart and respiratory diseases may occur. Patients usually die from the disease in their 20s or 30s;
Vyondys 53 is approved under the Accelerated Approval Pathway, which provides for the approval of drugs for serious or life-threatening diseases and often provides a meaningful advantage over existing treatments. Approvals under this pathway can be based on well-controlled studies that show that the drug has an effect on alternative endpoints that reasonably have the potential to predict the clinical benefits of the patient (i.e., the patient's feelings or functions or whether they survive). While the company conducts clinical trials to validate the predicted clinical benefits, this approach enables patients to obtain promising new drugs earlier.
The accelerated approval of Vyondys 53 is based on an alternative endpoint of increased skeletal muscular dystrophy protein production observed in some patients treated with the drug.
FDA concluded that the data submitted by the applicant showed an increase in muscular dystrophy protein production, which reasonably predicted the clinical benefits of duxing muscular dystrophy, a patient with a proven mutagenic protein mutation that skips Vyondys 53. The clinical benefits of the drug, including improved motor function, have not yet been determined. In making this decision, the U.S. Food and Drug Administration considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease, and the lack of available treatment.
Vyondys 53 was evaluated in a two-part clinical study. The first part consisted of 12 diabetics, 8 of whom were treated with Vyondys 53 and 4 with placebo. The second part of the study was open-minded, involving 12 patients who participated in the first part of the study, as well as 13 other patients who had not previously been treated. In this study, myocardial disorder protein levels increased from an average of 0.10% of normal levels at baseline to 1.02% of normal levels after 48 weeks or more of medication.
As part of the accelerated approval process, the U.S. Food and Drug Administration requires the company to conduct clinical trials to confirm the clinical benefits of the drug. The ongoing study aims to assess whether Vyondys 53 can improve the motor function of patients with Duxing muscular dystrophy, where the muscular dystrophy protein gene has been shown to mutate and skip Vyondys 53. If the trial fails to prove clinical benefits, the U.S. Food and Drug Administration may file a lawsuit to revoke approval of the drug.
The most common side effects reported in clinical studies by participants who received Vyondys 53 were headache, fever, cough, vomiting, abdominal pain, cold symptoms (nasopharyngitis) and nausea. Patients treated with Vyondys53 experience allergic reactions, including rashes, fever, itching, urticaria, skin irritation (dermatitis) and peeling (peeling).
In addition, renal toxicity was observed in animals receiving golodirsen. Although renal toxicity was not observed in the clinical study of Vyondys 53, renal toxicity, including potentially fatal ngball nephritis, was observed after the application of some antisant oligonucleotides. Patients taking Vyondys 53 should monitor kidney function.
The FDA approved the application's fast tracking and priority review designation.
Vyondys 53 also received the title of Orphan Drug, which provides incentives to help and encourage the development of drugs to treat rare diseases.
addition, the manufacturer received a rare paediatric disease priority review certificate. The FDA's Priority Review Voucher Program for Rare Pediatric Diseases is designed to encourage the development of new drugs and biologics to prevent and treat rare diseases in children.Saletta Therapeutics in Cambridge, Massachusetts, received approval from Vyondys 53. （cyy123.com）