The Food and Drug Administration has issued an industry guidance for the development of drugs to treat a notoriously devastating neurodegenerative disease.
On Monday, the agency issued its final guidance for developing drugs for amyotrophic lateral sclerosis, or ALS, a disease that causes progressive muscle weakness and ultimately leads to premature death.
A few drugs currently have FDA approval to treat the disease, according to the ALS Association. These include Japanese drugmaker Mitsubishi Tanabe Pharma’s Radicava (edaravone), which was the first new drug specifically for ALS in 22 years when the FDA approved it in 2017. Others include Avanir Pharmaceuticals’ Nuedexta (dextromethorphan hydrobromide/quinidien sulfate), used to treat pseudobulbar effect – in appropriate laughter or crying – that is a symptom of ALS, and various formulations of riluzole, which in its original form is now generic. Japan’s Otsuka Pharmaceutical acquired Avanir in 2014.
A projection in January by ResearchandMarkets.com forecast that the market for ALS drugs was expected to reach $1.2 billion during the 2017-2027 period — compared with drug sales of $187 million in 2017 — in the seven largest markets of the U.S., Japan and the top five countries in the European Union. The launch of six late-stage pipeline products will drive the growth, the report noted. One drug currently in development for ALS is Biogen’s BIIB067 (tofersen), which is in a Phase III study of 144 patients who have a mutation known as SOD1.
In terms of trial design, the FDA guidance emphasized that ALS trials should not randomize patients to only receive placebo, and that all patients in trials of drugs to treat the disease should receive the best standard of care.
“Various strategies can be applied to expedite ALS trials and minimize unnecessary exposure to placebo,” the guidance read. These include master protocols, which allow for the simultaneous evaluation of multiple drugs, with a common or shared placebo group. Adaptive designs and enrichment strategies should also be considered, the guidance stated.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
In trials’ measurement of the efficacy of ALS drugs, the guidance suggests that study endpoints should be based measures like the ALS Functional Rating Scale-Revised or similar scales, and assessments of effectiveness outcomes in trials like patient-reported outcomes should be included in addition to the primary endpoint.
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