A Neurology Pipeline for the Ages

Stephen D. Silberstein, MD

As 2019 comes to a close, I feel it’s important to reflect on breakthroughs and advances as it can help invigorate us and our practice in the new year. The past year saw dozens of drug approvals for common, well-known neurological disorders, such as headache and multiple sclerosis (MS), as well as lesser-known but serious disorders, including spinal muscular atrophy. Indeed, the neurology pipeline has been especially fruitful this year, and we have much to look forward to in 2020.

As a headache specialist, 2019 proved especially exciting as several new treatments have become available, including traditional pharmaceuticals as well as noninvasive devices. Notably, the FDA approved galcanezumab as the first drug for the treatment of episodic cluster headache, an incredibly disabling headache disorder with an extremely high unmet treatment need. The agency also recently approved lasmiditan, the first drug in a new class of migraine therapies aimed at optimizing acute relief. The specialty has also seen a surge in new technologies for the treatment of migraine headache, including wearables such as Nerivio, a smartphone-controlled device worn on the arm that promotes migraine pain relief through a conditioned pain response.

However, many other groundbreaking drugs entered other therapeutic areas over the course of the year. In MS, for instance, the FDA approved 2 new drugs just days apart— siponimod, which was the first oral drug approved for the treatment of active secondary-progressive MS as well as relapsing forms of the disease, was quickly followed by the approval of oral cladribine, which is also indicated for the treatment of active secondary-progressive disease and well as relapsing MS. Ongoing pursuits in immunotherapy for MS have also made an impact in 2019, with very promising data emerging from recent clinical trials of ofatumumab, as well as other therapeutic targets, including the S1P receptor modulator ponesimod. I look forward to following the research into 2020 knowing that more treatment choices will benefit both the clinician and the patient.

Perhaps one of the most intriguing developments of the year arrived in October, when Biogen and Eisai announced that they would put their anti-amyloid drug aducanumab in front of the FDA despite shelving the investigational therapy earlier in the year after a disappointing futility analysis. The companies announced that additional analysis of an expanded dataset revealed that patients who received the higher-dose drug for longer showed statistically significant improvements on a clinical dementia rating scale. Although clinical skepticism is especially high when looking at treatments for Alzheimer disease and dementia, I know that I am one of many who hope that the data is strong enough to support regulatory review and approval.

So much awaits us in 2020. I hope that you will stay with NeurologyLive® as your source for the latest updates in neurology, as well as key insights on topics that matter to you and your practice.