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Human Drugs

Harmony Reports Disappointing Fragile X Data

Harmony Biosciences reports topline results from a Phase 3 registrational clinical trial of ZYN002 in Fragile X syndrome did not meet the primary endp...

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Biologics

Cell/Gene Therapy Postapproval Monitoring Guide

FDA posts a draft guidance outlining methods drugmakers should use to collect long-term safety and efficacy data once cell and gene therapy products a...

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Biologics

Cellebration Refusing to Provide Records to FDA

FDA warns San Juan, Puerto Rico-based Cellebration LLC that it is violating the Federal Food, Drug, and Cosmetic Act and agency regulations by refusin...

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Human Drugs

Acadias Phase 3 Trial of Intranasal Carbetocin Fails

Acadia Pharmaceuticals says it will cease developing intranasal carbetocin after its Phase 3 COMPASS PWS trial of the drug for treating hyperphagia in...

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Biologics

Regenerative Therapies Expedited Programs

FDA publishes a draft guidance with recommendations for expedited development and review of regenerative medicine therapies for serious conditions.

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Biologics

CGT Trial Innovative Designs Guidance

FDA publishes a draft guidance on agency requirements and considerations for using various clinical trial designs and endpoints to support licensure o...

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Human Drugs

Interchangeable Biosimilar Bill Introduced

Reps. Pfluger and Landsman introduce bipartisan legislation to deem biosimilars to be interchangeable upon FDA approval.

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Human Drugs

uniQure Reports Favorable Data in Huntingtons Trial

uniQure reports favorable data on its investigational gene therapy AMT-130 in Huntingtons disease patients.

Biologics

CGMP Violations at Janssen Vaccines S. Korea Plant

FDA warns Janssen Vaccines about CGMP violations in its Incheon, South Korea, drug manufacturing facility.

Human Drugs

CGMP Deviations in Chengdu Records Review

FDA warns Sichuan, China-based Chengdu Brilliant Biopharmaceutical Co. about CGMP deviations in its manufacturing of active pharmaceutical ingredients...