Update: FDA approval of Biogen's aducanumab

Mechanical circulatory support for end-stage heart failure has become routine and is now increasingly used as definitive treatment. Several small devices qualify for this purpose, but only a few have gained US Food and Drug Administration (FDA) approval as yet. Several studies, including the Randomized Evaluation of Mechanical Assistance for the Treatment of Congestive Heart Failure (REMATCH) study, the Investigation of Non-transplant-Eligible Patients Who Are Inotrope Dependent (INTrEPID) and the HeartMate (HM) II trial have confirmed a significantly improved quality of life and functional capacity after device placement. However, cerebrovascular events, infection and device malfunction still pose a considerable risk to patients and hinder widespread use.

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FDA Approvals and Consensus Guidelines for Botulinum Toxins in the Treatment of Dystonia

Toxins ◽

10.3390/toxins12050332 ◽

2020 ◽

Vol 12 (5) ◽

pp. 332 ◽

Cited By ~ 2

Author(s):

Lauren L. Spiegel ◽

Jill L. Ostrem ◽

Ian O. Bledsoe

Keyword(s):

Botulinum Toxin ◽

Literature Review ◽

American Academy ◽

Practice Guidelines ◽

European Federation ◽

Botulinum Toxins ◽

Consensus Guidelines ◽

Off Label ◽

Fda Approval ◽

American Academy Of Neurology

In 2016, the American Academy of Neurology (AAN) published practice guidelines for botulinum toxin (BoNT) in the treatment of blepharospasm, cervical dystonia, adult spasticity, and headache. This article, focusing on dystonia, provides context for these guidelines through literature review. Studies that led to Food and Drug Administration (FDA) approval of each toxin for dystonia indications are reviewed, in addition to several studies highlighted by the AAN guidelines. The AAN guidelines for the use of BoNT in dystonia are compared with those of the European Federation of the Neurological Societies (EFNS), and common off-label uses for BoNT in dystonia are discussed. Toxins not currently FDA-approved for the treatment of dystonia are additionally reviewed. In the future, additional toxins may become FDA-approved for the treatment of dystonia given expanding research in this area.

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Repurposing existing drugs for new uses: a cohort study of the frequency of FDA-granted new indication exclusivities since 1997

Journal of Pharmaceutical Policy and Practice ◽

10.1186/s40545-020-00282-8 ◽

2021 ◽

Vol 14 (1) ◽

Author(s):

Babak Sahragardjoonegani ◽

Reed F. Beall ◽

Aaron S. Kesselheim ◽

Aidan Hollis

Keyword(s):

Therapeutic Potential ◽

Drug Repurposing ◽

Status Quo ◽

New Drugs ◽

Generic Entry ◽

Small Molecule Drugs ◽

Clinical Investigations ◽

Fda Approval ◽

Limited Duration ◽

Observation Period

Abstract Background Drug repurposing (i.e., finding novel uses for existing drugs) is essential for maximizing medicines’ therapeutic utility, but obtaining regulatory approval for new indications is costly. Policymakers have therefore created temporary indication-specific market exclusivities to incentivize drug innovators to run new clinical investigations. The effectiveness of these exclusivities is poorly understood. Objective To determine whether generic entry impacts the probability of new indication additions. Methods For a cohort of all new small-molecule drugs approved by the FDA between July 1997 and May 2020, we tracked new indications added for the subset of drugs that experienced generic entry during the observation period and then analyzed how the probability of a new indication changed with the number of years since/to generic entry. Results Of the 197 new drugs that subsequently experienced generic entry, only 64 (32%) had at least one new indication added. The probability of a new indication addition peaked above 4% between 7 and 8 years prior to generic entry and then to dropped to near zero 15 years after FDA approval. We show that the limited duration of exclusivity reduces the number of secondary indications significantly. Conclusion Status quo for most drug innovators is creating novel one-indication products. Despite indication-specific exclusivities, the imminence of generic entry still has a detectable impact on reducing the chances of new indication additions. There is much room for improvement when it comes to incentivizing clinical investigations for new uses and unlocking existing medicines’ full therapeutic potential.

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