The approvals introduced several new agents into a number of patient populations, including much-needed additions to some diseases lacking a large number of options.
2020 has been a monumental year for new therapies for neurologic diseases. From sleep disorders to neuromuscular disease, the FDA has approved numerous new treatments to address unmet needs in a range of disorders, including the first-ever oral therapy for spinal muscular atrophy, as well as a new intranasal rescue formulation for seizure activity.
FDA actions are a foundational part of the NeurologyLive team's coverage each year, as many of these approvals marked important moments for a number of patient populations and their health care professionals. Any addition to the therapeutic landscape of a disease is a welcome sign, as each new therapy provides a new approach to care.
Look back on some of the most significant FDA approvals in 2020 by clicking the buttons below:
Approved in January 2020, intranasal diazepam (Valtoco; Neurelis), which is a schedule IV controlled substance, is the first nasal spray approved as rescue medication in patients 6 years and older with epilepsy.
Rimegepant (Nurtec ODT; Biohaven), the only orally disintegrating anti-CGRP tablet is available in a 75-mg dose and was the company’s first agency approval.
The once-daily, oral, selective catechol-O-methyltransferase (COMT) inhibitor opicapone (Ongentys; Neurocrine Biosciences) helps increase ON time without promoting dyskinesia.
The novel fumarate bioequivalent, monomethyl fumerate (Bafiertam; Banner Life Science) is an alternative to Tecfidera (dimethyl fumarate; Biogen), and enters the market as a new oral treatment option for relapsing forms of multiple sclerosis.
Riddiplam (Evrysdi; PTC Therapeutics), is the first at-home therapy indicated for the treatment of adults and children with SMA, approved in a liquid formulation for patients over the aged 2 months and older.
Approved in late August, the anti-CD20 monoclonal antibody ofatumumab (Kesimpta; Novartis) is administered once monthly at home and is available for adults with multiple sclerosis (MS), including clinically isolated syndrome (CIS), relapsing-remitting disease, and active secondary progressive MS.
Pitolisant (Wakix; Harmony Biosciences) was previously approved for the treatment of excessive daytime sleepiness in adults with narcolepsy and is the only FDA-approved treatment for both excessive daytime sleepiness and cataplexy in narcolepsy that is not a scheduled controlled substance
This latest FDA indication for lacosamide (Vimpat; UCB) includes adjunctive therapy for those aged 4 years and older in both the CV and intravenous formulations of the product.