Le Lézard
Classified in: Health, Science and technology
Subject: FDA

FDA Approves New Treatment for Pompe Disease


SILVER SPRING, Md., Aug. 6, 2021 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease.

Patients with Pompe disease have an enzyme deficiency that leads to the accumulation of a complex sugar, called glycogen, in skeletal and heart muscles, which cause muscle weakness and premature death from respiratory or heart failure. Normally, glycogen?the stored form of glucose?breaks down to release glucose into the bloodstream to be used as fuel for the cells.

"Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people's lives," said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA's Center for Drug Evaluation and Research. "Today's approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for rare diseases, including Pompe disease."  

Nexviazyme, an enzyme replacement therapy, is an intravenous medication that helps reduce glycogen accumulation. The effectiveness of Nexviazyme for the treatment of Pompe disease was demonstrated in a study of 100 patients who were randomized to take Nexviazyme or another FDA-approved enzyme replacement therapy for Pompe disease. Treatment with Nexviazyme improved lung function similar to the improvement seen with the other therapy.

The most common side effects included headache, fatigue, diarrhea, nausea, joint pain (arthralgia), dizziness, muscle pain (myalgia), itching (pruritus), vomiting, difficulty breathing (dyspnea), skin redness (erythema), feeling of "pins and needles" (paresthesia) and skin welts (urticaria). Serious reactions included hypersensitivity reactions like anaphylaxis and infusion-associated reactions, including respiratory distress, chills and raised body temperature (pyrexia). Patients susceptible to fluid volume overload or with compromised cardiac or respiratory function may be at risk for serious acute cardiorespiratory failure.

The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations. Nexviazyme also received an orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Nexviazyme to Genzyme Corporation.

Media Contact: Alison Hunt, 202-308-5496
Consumer Inquiries: Email or 888-INFO-FDA

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

U.S. Food and Drug Administration (FDA) logo (PRNewsfoto/FDA)

 

SOURCE U.S. Food and Drug Administration


These press releases may also interest you

15 oct 2021
Helix BioPharma Corp. ("Helix" or the "Company"), an immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, announced today that it anticipates the filing of its annual financial...

15 oct 2021
The Centers for Medicare and Medicaid Services (CMS) released its annual quality rankings. For the 2022 plan year, Network Health earned a 5 out of 5-Star Rating from CMS for its Medicare Advantage PPO plans. This is Medicare's highest possible...

15 oct 2021
Boehringer Ingelheim today announced the U.S. Food and Drug Administration (FDA) approved the supplemental Biologics License Application (sBLA) for Cyltezo® (adalimumab-adbm) as the first Interchangeable biosimilar with Humira® (adalimumab). The FDA...

15 oct 2021
Novartis Pharmaceuticals Canada Inc. applauds the government of Quebec's decision to provide public reimbursement for Zolgensma® (onasemnogene abeparvovec)...

15 oct 2021
Worcester Polytechnic Institute (WPI) researcher Loris Fichera has developed a flexible, slimmed-down robotic laser probe for endoscopic larynx surgeries that could reduce recovery time for patients and cut health care spending by shifting procedures...

15 oct 2021
The "Global Industrial Radiography Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Technique (Film-based Radiography and Digital Radiography); End-User Industry" report has been added to ResearchAndMarkets.com's offering. The...



News published on 6 august 2021 at 10:54 and distributed by: