FDA approves first treatment for inherited rare disease

FDAToday, the U.S. Food and Drug Administration granted approval to Givlaari (givosiran) for the treatment of adult patients with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood).

"This buildup can cause acute attacks, known as porphyria attacks, which can lead to severe pain and paralysis, respiratory failure, seizures and mental status changes. These attacks occur suddenly and can produce permanent neurological damage and death," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research. "Prior to today's approval, treatment options have only provided partial relief from the intense unremitting pain that characterizes these attacks. The drug approved today can treat this disease by helping to reduce the number of attacks that disrupt the lives of patients."

The approval of Givlaari was based on the results of a clinical trial of 94 patients with acute hepatic porphyria. Patients received a placebo or Givlaari. Givlaari’s performance was measured by the rate of porphyria attacks that required hospitalizations, urgent health care visits or intravenous infusion of hemin at home. Patients who received Givlaari experienced 70% fewer porphyria attacks compared to patients receiving a placebo.

Common side effects for patients taking Givlaari were nausea and injection site reactions. Health care professionals are advised to monitor patients for anaphylactic (allergic) reaction and renal (kidney) function. Patients should have their liver function tested before and periodically during treatment.

The FDA granted this application Breakthrough Therapy designation and Priority Review designation. Givlaari also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Givlaari to Alnylam Pharmaceuticals.

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.

Most Popular Now

SK bioscience and GSK start Phase 3 trial of adjuv…

SK bioscience (SK) and GlaxoSmithKline plc (GSK) today announced the initiation of a Phase 3 clinical study of SK's COVID-19 vaccine candidate, GBP510, in combination wit...

Blood vessels produce growth factor that promotes …

Blood vessels supply tumors with nutrients and, on the other hand, enable cancer cells to spread throughout the body. The settlement of circulating tumor cells in a dista...

No serious health effects linked to mRNA COVID-19 …

Federal and Kaiser Permanente researchers combing the health records of 6.2 million patients found no serious health effects that could be linked to the 2 mRNA COVID-19 v...

First-in-human clinical trial for a vaccine to tre…

The first patients have been enrolled in a phase 1 randomized placebo-controlled clinical trial to study a therapeutic vaccine for opioid use disorder developed by resear...

New study examines 'Achilles heel' of cancer tumou…

Researchers at the University of British Columbia's faculty of medicine and BC Cancer Research Institute have uncovered a weakness in a key enzyme that solid tumour cance...

AI algorithm solves structural biology challenges

Determining the 3D shapes of biological molecules is one of the hardest problems in modern biology and medical discovery. Companies and research institutions often spend ...

A drug costing less than €2 a day helps in the tre…

Metoprolol, a drug widely used to treat cardiovascular disease, is beneficial when administered to COVID-19patients. This is the finding of a study by investigators at th...

Sandoz strengthens pipeline by entering into agree…

Sandoz, a Novartis division, today announced that it has entered into a commercialization agreement with Bio-Thera Solutions, Ltd. for biosimilar bevacizumab (BAT1706). B...

Pfizer and BioNTech submit a variation to EMA with…

Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) announced that they submitted a variation to the European Medicines Agency (EMA) requesting to update the Condition...

Rheumatoid arthritis treated with implanted cells …

With a goal of developing rheumatoid arthritis therapies with minimal side effects, researchers at Washington University School of Medicine in St. Louis have genetically ...

One in three Americans had COVID-19 by the end of …

A new study published in the journal Nature estimates that 103 million Americans, or 31 percent of the U.S. population, had been infected with SARS-CoV-2 by the end of 20...

NIH scientists build a cellular blueprint of multi…

Chronic lesions with inflamed rims, or "smoldering" plaques, in the brains of people with multiple sclerosis (MS) have been linked to more aggressive and disabling forms ...