FDA approves new dosage form for the treatment of amyotrophic lateral sclerosis (ALS)
On Nov. 22, 2019, the U.S. Food and Drug Administration (FDA) approved ExservanTM (riluzole) oral film for the treatment of amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig’s Disease. ALS is a progressive disease that affects nerve cells in the brain and spinal cord that eventually results in muscle weakness, and oftentimes leads to paralysis. Riluzole was originally approved by the FDA under the trade names of Rilutek® (1995) and TiglutikTM (2018) as an oral tablet and oral suspension, respectively. ExservanTM, as an orally dissolvable film, presents a new treatment option for ALS patients that may face difficulties swallowing or using traditional medication forms, such as tablets and suspensions. According to the ALS Association, there are a roughly 5,000 people diagnosed with the disease each year.
FDA issues warning letters to 15 companies for illegal sale of cannabidiol (CBD) products
On Nov. 25, 2019, the U.S. FDA announced it had sent warning letters to 15 companies for the illegal sale of CBD products. Violations identified by the FDA include advertising various CBD products as dietary supplements and adding CBD to human foods, such as chocolate bars and teas, as well as animal foods. In addition, the FDA published a revised Consumer Update that details further safety concerns surrounding CBD products, including possible liver injury, drug interactions and changes in mood. The update clarifies that, based on existing data, the FDA cannot conclude that CBD is generally recognized as safe (GRAS) for use in animal or human food. The FDA plans to provide further guidance regarding their regulatory approach to these products in the coming weeks.
From the Industry
Phase III trial shows positive results for oral migraine medication
On Nov. 19, 2019, the Journal of the American Medical Association (JAMA) released results of the ACHIEVE II clinical trial of ubrogepant, an oral medication being studied for migraine treatment. Ubrogepant belongs to a class of medications known as calcitonin-gene-related peptide (CGRP) receptor antagonists, which are currently available on the market only as injectables and indicated for migraine prevention, rather than migraine treatment. The trial showed a higher percentage of patients achieved pain relief with ubrogepant 50 mg and 25 mg when compared to placebo. More patients also experienced relief from their most bothersome symptom with 50 mg urbogepant than 25 mg ubbrogepant or placebo. The rate of adverse effects was similar in all study groups, indicating good safety and tolerability of the drug.
Recent FDA Approvals
Novel Drug Approval: GivlaariTM (givosiran)
Subcutaneous injection for the treatment of adult patients with acute hepatic porphyria; a rare blood disorder [11/20/2019 – Orphan Drug; Priority Review – ALNYLAM PHARMS INC]
Novel Drug Approval: Xcopri® (cenobamate)
Oral tablet for the treatment of partial-onset seizures in adult patients [11/21/2019 – SK LIFE SCIENCE INC]
Novel Drug Approval: OxbrytaTM (voxelotor)
Oral tablet for the treatment of sickle cell disease in patients aged 12 years and older [11/25/2019 – Accelerated Approval; Fast Track; Orphan Drug; Priority Review – GLOBAL BLOOD THERAPEUTICS INC]
New Generics Entering the Marketplace
Indication: Ulcerative Colitis
Dosage Form/Strength: 0.375g
Average Wholesale Price (AWP): Generic = $551 | Brand = $612