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AbbVie Neurodegenerative Disorder Drug Gets Orphan Status - Analyst Blog

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AbbVie Inc.ABBV and C 2 N Diagnostics announced that the FDA has granted C 2 N-8E12 (ABBV-8E12) orphan drug status for the treatment of patients suffering from progressive supranuclear palsy (PSP; which is also known as Steele-Richardson-Olszewski syndrome).

We note that the FDA usually grants orphan drug status to novel drugs or biologics being developed as a potential treatment for rare diseases/conditions that affect less than 200,000 people in the U.S. Orphan drug status is also granted to drugs addressing diseases afflicting more than 200,000 people but these drugs are not expected to recover the cost of developing and marketing the same. However, this designation does not alter the standards of regulatory requirements and process for obtaining marketing approval for the candidate. Safety and efficacy of the compound for the treatment of a disease must be established through adequate and well-controlled studies.

C 2 N-8E12 (ABBV-8E12), a recombinant humanized anti-tau antibody, is currently in a randomized, double-blind, placebo-controlled, single ascending dose, multi-center phase I study. The study, which is estimated to involve 32 patients with PSP, is being conducted to evaluate the safety, tolerability and pharmacokinetics of the candidate.

We remind investors that AbbVie has an exclusive worldwide license agreement with C 2 N Diagnostics for the development and commercialization of a portfolio of anti-tau antibodies for the treatment of PSP, Alzheimer's disease and other neurological disorders entered into earlier this year.

We are encouraged by the FDA granting orphan drug status to C 2 N-8E12 (ABBV-8E12). As per information provided by AbbVie, PSP, a progressive neurodegenerative disorder, affects about 20,000 individuals in the U.S. with an estimated annual incidence of one per 100,000 people above 60 years of age. With no treatments currently being approved for PSP, there is an unmet need for drugs addressing PSP.

AbbVie currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Agios Pharmaceuticals, Inc. AGIO , AMAG Pharmaceuticals, Inc. AMAG and Eleven Biotherapeutics, Inc. EBIO . All three hold a Zacks Rank #1 (Strong Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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