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

Novartis and life sciences companies commit expert…

Novartis and a consortium of life sciences companies announced an important collaboration to accelerate the development, manufacture and delivery of vaccines, diagnostics...

Amgen and the Amgen Foundation commit up to $12.5 …

Amgen (NASDAQ:AMGN) and the Amgen Foundation announced an initial commitment of up to $12.5 million to support U.S. and global relief efforts to address critical needs in...

AstraZeneca to donate 9 million face masks to supp…

AstraZeneca is donating nine million face masks to support healthcare workers around the world as they respond to the COVID-19 (novel coronavirus) global pandemic. AstraZ...

Roche response to COVID-19 pandemic

Roche Group (SIX: RO, ROG; OTCQX: RHHBY), provided an update on the various actions the company is taking to address the COVID-19 pandemic. On March 19, 2020, Roche co...

Singapore modelling study estimates impact of phys…

A new modelling study conducted in a simulated Singapore setting has estimated that a combined approach of physical distancing [2] interventions, comprising quarantine (f...

Consignment of 30 million hydroxychloroquine table…

Novartis announced that Sandoz, its generics and biosimilars division, has started to ship a consignment of 30 million doses of hydroxychloroquine tablets to the US Depar...

CAR macrophages go beyond T cells to fight solid t…

Chimeric antigen receptor (CAR) T cell therapy has been a game-changer for blood cancers but has faced challenges in targeting solid tumors. Now researchers from the Pere...

FDA approves first plasma therapy for Houston Meth…

Houston Methodist received FDA approval Saturday to become the first academic medical center in the nation to transfuse donated plasma from a recovered COVID-19 patient i...

Experimental AI tool predicts which COVID-19 patie…

An artificial intelligence tool accurately predicted which patients newly infected with the COVID-19 virus would go on to develop severe respiratory disease, a new study ...

Vivli to launch a portal for sharing data from COV…

In a visible sign of data sharing leadership, Vivli, the Center for Clinical Research Data has committed to serving the open science community through the launch of a COV...

Roche initiates Phase III clinical trial of Actemr…

Roche (SIX: RO, ROG; OTCQX: RHHBY) is working with the Food & Drug Administration (FDA) to initiate a randomised, double-blind, placebo-controlled Phase III clinical tria...

Novartis commits to donate up to 130 million doses…

Novartis announced its commitment to donate up to 130 million doses of generic hydroxychloroquine to support the global COVID-19 pandemic response. Hydroxychloroquine and...