The FDA Center for Drug Evaluation and Research, Division of Neurology Products, regulates and reviews Investigational New Drug applications and marketing applications for drug and biologic products for the treatment of neurologic diseases and conditions. The diseases and conditions that are within the authority of the Division of Neurology Products include, but are not limited to, Alzheimer’s disease, stroke, amyotrophic lateral sclerosis (ALS), Huntington’s disease, epilepsy, Parkinson’s disease, migraine headaches, muscular dystrophy, multiple sclerosis, dementia, and narcolepsy.1
The review and approval by the FDA of drug and biologic products for treatment of neurologic disorders are critical in the context of public health. For example, 6.5% to 7.9% of adults in the United States are affected by a brain impairment.2 More specifically, the national prevalence of certain neurologic disorders in the U.S. is estimated to be 5.3 million cases of Alzheimer’s disease, 30,000 cases of ALS, 30,000 cases of Huntington’s disease, 2.3 million epilepsy cases, 1 million cases of Parkinson’s disease, 28 million cases of migraine headaches, 40 million cases of anxiety disorders, and 350,000 cases of multiple sclerosis, as well as 2.6% of adults with stroke.3
Streamlining Drug Development
As part of its regulatory authority related to approval of drugs and biologicals for the treatment of neurologic disorders, the FDA issued guidance documents during February 2018. The FDA published finalized guidance regarding clinical trials related to Duchenne muscular dystrophy and migraines and draft guidance documents for trials related to Alzheimer’s disease, ALS, and pediatric seizures. The agency intended for the documents to provide details on how researchers may best approach drug development for certain neurologic conditions. The FDA expressed the hope that providing up-to-date, clear information about its scientific expectations would save companies time and resources and, ultimately, bring effective new medicines to patients more efficiently.4
The issuance of the guidance documents was followed by a number of novel drug approvals by the FDA of drugs for the treatment or management of certain neurologic conditions, including:
• Aimovig (erenumab-aooe) by Amgen, Inc. for preventive treatment of migraines in adults (May 2018)
• Epidiolex (cannabidiol) by GW Research, Ltd. for the treatment of rare, severe forms of epilepsy (June 2018)
• Onpattro (patisiran sodium) by Alnylam Pharmaceuticals, Inc. for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (August 2018)
• Diacomit (stiripentol) by BioCodex for the treatment of seizures associated with Dravet syndrome in patients aged 2 years and older taking clobazam (August 2018)
• Emgality (galcanezumab-gnlm) by Eli Lilly and Company for the preventive treatment of migraines in adults (September 2018)
• Ajovy (fremanezumab-vfrm) by Teva Pharmaceuticals USA, Inc. for the preventive treatment of migraines in adults (September 2018)
• Tegsedi (inotersen sodium) by Ionis Pharmaceuticals, Inc. for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (October 2018).5
Advancing Migraine, Seizure Treatments
These drug approvals represent significant advances in the treatment of neurologic disorders. Aimovig, for example, is the first FDA-approved preventive migraine treatment in a new class of drugs that work by blocking the activity of calcitonin gene–related peptide, a molecule that is involved in migraine attacks. Epidiolex is the first FDA-approved cannabidiol product for treatment of Dravet and Lennox-Gastaut syndromes. Onpattro is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating, and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart, and other organs. Onpattro is also the first FDA approval of a new class of medications called small interfering ribonucleic acid treatment.6
These drug approvals and guidance documents are indicative of the FDA’s approach in streamlining the administrative process for bringing new drugs to market, all of which should benefit the patient.
1. FDA. Office of Drug Evaluation I–Division of Neurology Products (DNP). www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm290674.htm. Accessed December 2, 2018.2. Family Caregiver Alliance. Incidence and prevalence of the major causes of brain impairment. www.caregiver.org/incidence-and-prevalence-major-causes-brain-impairment. Accessed December 2, 2018.3. OHSU Brain Institute. www.ohsu.edu/xd/health/services/brain/in-community/brain-awareness/brain-health/disease-statistics.cfm. Accessed December 2, 2018.4. FDA. Early Alzheimer’s disease: developing drugs for treatment–guidance for industry. Draft February 2018. www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM596728.pdf. Accessed December 2, 2018; See also: FDA. Amyotrophic lateral sclerosis: developing drugs for treatment–guidance for industry. Draft February 2018. www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM596718.pdf. Accessed December 2, 2018; FDA. Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment–guidance for industry. February 2018. www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm450229.pdf. Accessed December 2, 2018; FDA. Drugs for treatment of partial onset seizures: full extrapolation of efficacy from adults to pediatric patients 4 years of age and older–guidance for industry. February 2018. www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM596731.pdf. Accessed December 2, 2018; FDA. Migraine: developing drugs for acute treatment–guidance for industry. www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM419465.pdf. Accessed December 2, 2018; Statement from FDA Commissioner Scott Gottlieb, M.D. on advancing the development of novel treatments for neurological conditions; part of broader effort on modernizing FDA’s new drug review programs. February 15, 2018. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm596897.htm. Accessed December 3, 2018.5. FDA. Novel drug approvals for 2018. www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm592464.htm. Accessed December 3, 2018. See also: Aquestive Therapeutics announces U.S. Food and Drug Administration (FDA) approval for SYMPAZAN (clobazam) Oral Film. November 2, 2018. www.prnewswire.com/news-releases/aquestive-therapeutics-announces-us-food-and-drug-administration-fda-approval-for-sympazan-clobazam-oral-film-300742913.html. Accessed December 3, 2018; See also: PERSERIS, the first once-monthly risperidone-containing subcutaneous long-acting injectable, now available in the U.S. for the treatment of schizophrenia in adults. November 19, 2018. www.indivior.com/wp-content/uploads/2018/11/Perseris-availability.pdf. Accessed December 3, 2018.6. FDA News Release. FDA approves novel preventive treatment for migraine. May 17, 2018. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm608120.htm Accessed December 3, 2018; See also: Epidiolex. www.epidiolex.com. Accessed December 3, 2018; FDA News Release. FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616518.htm. Accessed December 3, 2018.
The information in this article is general in nature and is not intended to provide legal or other professional advice.
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