Pharma

ALS disrupted: FDA approves Japanese drug that slows decline by 33%

Late Friday, MT Pharma America broke the news that the FDA had approved its experimental amyotrophic lateral sclerosis (ALS) drug Radicava, the second-ever drug approved for ALS and the first in over 20 years.

ALS single letters pinned on cork noticeboard

Late Friday, MT Pharma America broke the news that the FDA had approved its experimental amyotrophic lateral sclerosis (ALS) drug Radicava (edaravone), the second-ever drug approved for ALS and the first in over 20 years.

Also known as Lou Gehrig’s disease, ALS is a rapidly progressive neurodegenerative disease that affects 12,000-15,000 Americans, according to the CDC. A majority of patients die within two-to-five years of diagnosis.

To be clear, this isn’t a modified version of an existing drug. Nor is it designed to simply treat the symptoms. In a pivotal Phase 3 study evaluating 137 patients with ALS, those receiving Radicava for six months experienced a 33 percent reduction in their rate of decline, compared to those taking the standard of care (Rilutek). 

While the exact mechanism of action is not yet known, the drug appears to protect motor neurons in the brain, noted Jean Hubble, MT Pharma’s VP of medical affairs.

“We have preclinical studies that would suggest that it reduces oxidative injury to the motor cells, the motor neurons,” Hubble said via phone. “Those are the cells in the brain and spinal cord that are responsible for sending signals for all our voluntary movements.”

Untreated, the loss of motor neurons leads to difficulty carrying out basic tasks such as buttoning up a shirt, walking, talking, and ultimately, even breathing.

Given the urgent need for a therapy in this space, FDA took the unusual measure of approving the drug on the back of a six-month study in Japan. Additional clinical trials in the United States were bypassed, seemingly to get the drug into patients’ hands.

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” said Eric Bastings, deputy director of neurology products at the FDA’s Center for Drug Evaluation and Research, in an agency statement.

To underscore how unusual the agency’s decision was, the drug hadn’t been allowed into the country until today. With its development resources fully committed to Japan, neither MT Pharma nor its parent company Mitsubishi Tanabe Pharma Corporation had filed an investigational new drug (IND) application in the United States. 

Fortunately for patients, the company has been stockpiling the drug overseas.

“Because urgency is so critical here, we actually started manufacturing the product at risk about six months ago and we have produced hundreds of thousands of IV bags,” said Tom Larson, MT Pharma’s chief commercial officer.

As a result of the company’s calculated bet, patients in the United States could have access to the drug by early to mid-August, Larson stated.

The drug is administered via infusion, a process that lasts around one hour. Patients cycle through two weeks of daily therapy, then two weeks off. This was done in a hospital setting during the trials in Japan, but Larson anticipates U.S. patients will eventually be able to take the infusion at home.

While the pivotal study only lasted six months (due to Japanese regulations), Hubble said data from open-label extension studies give no indication that the drug loses efficacy over time. It has been observed that patients who begin the treatment earlier tend to fare better long term, she said.

The status of post-approval studies or data requirements hasn’t yet been set, but as of Friday, Hubble was confident that no additional placebo-controlled studies will be needed.

In the meantime, there’s a lot to celebrate at the Jersey City, New Jersey headquarters of MT Pharma.

“It’s just remarkably gratifying and humbling to have this chance to make a difference in the lives of these individuals,” said Hubble, who started her career on the frontlines as a neurologist.

Photo: oko_swanomurphy, Getty Images 

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