FDA approves first drug for spinal muscular atrophy

FDAThe U.S. Food and Drug Administration has approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. Spinraza is an injection administered into the fluid surrounding the spinal cord.

"There has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life," said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. "As shown by our suggestion to the sponsor to analyze the results of the study earlier than planned, the FDA is committed to assisting with the development and approval of safe and effective drugs for rare diseases and we worked hard to review this application quickly; we could not be more pleased to have the first approved treatment for this debilitating disease."

SMA is a hereditary disease that causes weakness and muscle wasting because of the loss of lower motor neurons controlling movement. There is wide variability in age of onset, symptoms and rate of progression. Spinraza is approved for use across the range of spinal muscular atrophy patients.

The FDA worked closely with the sponsor during development to help design and implement the analysis upon which this approval was based. The efficacy of Spinraza was demonstrated in a clinical trial in 121 patients with infantile-onset SMA who were diagnosed before 6 months of age and who were less than 7 months old at the time of their first dose. Patients were randomized to receive an injection of Spinraza, into the fluid surrounding the spinal cord, or undergo a mock procedure without drug injection (a skin prick). Twice the number of patients received Spinraza compared to those who underwent the mock procedure. The trial assessed the percentage of patients with improvement in motor milestones, such as head control, sitting, ability to kick in supine position, rolling, crawling, standing and walking.

The FDA asked the sponsor to conduct an interim analysis as a way to evaluate the study results as early as possible; 82 of 121 patients were eligible for this analysis. Forty percent of patients treated with Spinraza achieved improvement in motor milestones as defined in the study, whereas none of the control patients did.

Additional open-label uncontrolled clinical studies were conducted in symptomatic patients who ranged in age from 30 days to 15 years at the time of the first dose, and in presymptomatic patients who ranged in age from 8 days to 42 days at the time of first dose. These studies lacked control groups and therefore were more difficult to interpret than the controlled study, but the findings appeared generally supportive of the clinical efficacy demonstrated in the controlled clinical trial in infantile-onset patients.

The most common side effects found in participants in the clinical trials on Spinraza were upper respiratory infection, lower respiratory infection and constipation. Warnings and precautions include low blood platelet count and toxicity to the kidneys (renal toxicity). Toxicity in the nervous system (neurotoxicity) was observed in animal studies.

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The sponsor is receiving a rare pediatric disease priority review voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive priority review of a subsequent marketing application for a different product. This is the eighth rare pediatric disease priority review voucher issued by the FDA since the program began.

Spinraza is marketed by Biogen of Cambridge, Massachusetts and was developed by Ionis Pharmaceuticals of Carlsbad, California.

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

Sanofi and Regeneron announce FDA Approval of Kevz…

Sanofi and Regeneron Pharmaceuticals, Inc. have announced the U.S. Food and Drug Administration (FDA) approval of Kevzara® (sarilumab) for the treatment of adult patients...

Read more

FDA approves first cancer treatment for any solid …

The U.S. Food and Drug Administration today granted accelerated approval to a treatment for patients whose cancers have a specific genetic feature (biomarker). This is th...

Read more

FDA approves drug to treat ALS

The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig's...

Read more

AstraZeneca marks a key milestone with the ‘toppin…

AstraZeneca marks a key milestone in its successful move to Cambridge, UK, with the 'topping out' of its new, state-of-the-art, strategic R&D centre and global corporate ...

Read more

Imfinzi significantly reduces the risk of disease …

AstraZeneca and MedImmune, its global biologics research and development arm, today announced positive results for the Phase III PACIFIC trial, a randomised, double-blind...

Read more

Abbott announces CE Mark and first use of the worl…

Abbott (NYSE: ABT) today announced CE Mark and first use of the new Confirm RxTM Insertable Cardiac Monitor (ICM), the world's first smartphone compatible ICM that will h...

Read more

Antibiotic doxycycline may offer hope for treatmen…

A study published in the journal Scientific Reports suggests that doxycycline, an antibiotic used for over half a century against bacterial infections, can be prescribed ...

Read more

Novartis exercises exclusive option agreement with…

Novartis announced today that it has notified Conatus Pharmaceuticals Inc., of its exercise of the option to an exclusive license for the global development and commercia...

Read more

High levels of exercise linked to nine years of le…

Despite their best efforts, no scientist has ever come close to stopping humans from aging. Even anti-aging creams can't stop Old Father Time. But new research from Brigh...

Read more

Vitamin A deficiency is detrimental to blood stem …

Many specialized cells, such as in the skin, gut or blood, have a lifespan of only a few days. Therefore, steady replenishment of these cells is indispensable. They arise...

Read more

Nearly 1 in 3 drugs found to have safety concerns …

How often are safety concerns raised about a drug after it's been approved by the FDA? Nicholas Downing, MD, of the Department of Medicine at Brigham and Women's Hospital...

Read more

Alzheimer's experts call for changes in FDA drug a…

Leading Alzheimer's disease researchers and a prominent patient advocate today published an analysis, "Single Endpoint for New Drug Approvals for Alzheimer's Disease," ur...

Read more

Pharmaceutical Companies

[ A | B | C | D | E | F | G | H | I | J | K | L | M | N | O | P | Q | R | S | T | U | V | W | X | Z ]