Experimental ALS Drug Gets Negative Review From FDA
Patients have been advocating for regulators to approve the drug from Amylyx Pharmaceuticals, but the Food and Drug Administration has issued a negative review, skeptical of its prospective benefits. Stat notes the FDA did mention "regulatory flexibility" when it comes to drug approval, however.
FDA Skeptical Of Benefits From Experimental ALS Drug
Federal health regulators issued a negative review Monday of a closely watched experimental drug for the debilitating illness known as Lou Gehrig’s disease, after months of lobbying by patient advocates urging approval. The drug from Amylyx Pharmaceuticals has become a rallying cause for patients with the deadly neurodegenerative disease ALS, their families and members of Congress who’ve joined in pushing the Food and Drug Administration to greenlight the drug. (Perrone, 3/28)
FDA Is Critical Of Data On Amylyx ALS Drug, But Cites 'Regulatory Flexibility'
Results from a single clinical trial of an experimental drug for ALS developed by Amylyx Pharmaceuticals “may not be sufficiently persuasive” to support approval, according to a review of the drug posted Monday by officials with the Food and Drug Administration. Reviewers in the FDA’s Office of Neuroscience criticized the data, which showed the drug demonstrated a modest slowing of disease progression but no survival benefit, as well as the conduct of the study. But the agency, in its review, noted that it can exercise “regulatory flexibility” to approve drugs for serious diseases with unmet medical needs. (Feuerstein, 3/28)
In other pharmaceutical news —
Men's Use Of Diabetes Drug Just Before Conception Is Linked To A 40% Increase In Birth Defects, Study Finds
Metformin use by men in the three-month period before they conceived a child was linked to a 40% higher risk of birth defects in the offspring, according to a study published Monday in the journal Annals of Internal Medicine. Metformin is a first-line drug in the treatment of type 2 diabetes. The research, which was done out of Denmark, used national registries to follow over 1 million births between 1997 and 2016 and compared the risk of major birth defects in babies based on paternal exposures to diabetes medications. The study observed only children who were born to women under 35 and men under 40. Babies born to women with diabetes were excluded. The researchers considered men exposed to metformin if they filled a prescription for it in the three months before conception, which is how long it takes the fertilizing sperm to fully mature. (Ahmed, 3/28)
Fragmented Regulations Exacerbate Saline Shortage
Different regulations for pharmaceuticals and medical devices are complicating the sodium chloride shortage. Hospitals have been dealing with intermittent shortages of sodium chloride and its related components since late last year, as manufacturers and distributors have been focused on COVID-19 vaccine production, some suppliers have pulled out of the market and those that remain have experienced production delays. About 60% of hospitals' requests for sodium chloride were not being filled as of Thursday, according to the group purchasing and consulting organization Premier. In normal times, less than 10% of hospital requests go unfulfilled. (Kacik, 3/28)
Louisville Courier Journal:
Kentucky Pharmacists Back Effort To Curb Prescription Drug Middlemen
A bill Kentucky pharmacists are seeking to curb the powers of prescription drug insurance middlemen appears stalled in the Senate after it sailed through the House March 21 on a vote of 88-3. House Bill 457, aimed at for-profit entities known as pharmacy benefit managers, or PBMs, has been consigned to the Senate Appropriations and Revenue Committee — often a dead-end for legislation. It follows a law the legislature enacted in 2020 cutting PBMs out of the state's $1.7 billion-a-year Medicaid prescription drug program amid complaints from pharmacists that PBMs were profiting at their expense. (Yetter, 3/28)