FDA opens up an ethical can of worms with accelerated approval for first drug for underlying genetic cause of dwarfism

Zachary Brennan

Senior Editor

The FDA on Friday signed off on an accelerated approval for BioMarin’s Voxzogo (vosoritide) injection, the first treatment to target the underlying genetics of dwarfism …

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FDA opens up an ethical can of worms with accelerated approval for first drug for underlying genetic cause of dwarfism

Zachary Brennan

Senior Editor

Sign up to read this article for free.

Among pharma CEOs, the IRA now and the IRA later get very different reactions

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Critical Junctures in Gene Therapy Clinical Material Manufacturing: Where Do I Start? And Will It Work?

FDA opens up an eth­i­cal can of worms with ac­cel­er­at­ed ap­proval for first drug for un­der­ly­ing ge­net­ic cause of dwarfism

Zachary Brennan

Senior Editor

Sign up to read this article for free.

Among phar­ma CEOs, the IRA now and the IRA lat­er get very dif­fer­ent re­ac­tions

Bris­tol My­ers restarts ra­dio­phar­ma­ceu­ti­cal Phase 3 tri­al, but de­lays read­out by one year

Up­dat­ed: With cost-cut­ting un­der­way, Bris­tol My­ers rais­es guid­ance, touts Phase 3 win and scraps an­oth­er

An­drew Left, short sell­er who cam­paigned against Valeant, is ac­cused of fraud by SEC

Eu­ro­pean drug reg­u­la­tor re­jects Alzheimer's drug Leqem­bi, cit­ing safe­ty con­cerns

Crit­i­cal Junc­tures in Gene Ther­a­py Clin­i­cal Ma­te­r­i­al Man­u­fac­tur­ing: Where Do I Start? And Will It Work?

FDA opens up an ethical can of worms with accelerated approval for first drug for underlying genetic cause of dwarfism

Among pharma CEOs, the IRA now and the IRA later get very different reactions

Bristol Myers restarts radiopharmaceutical Phase 3 trial, but delays readout by one year

Updated: With cost-cutting underway, Bristol Myers raises guidance, touts Phase 3 win and scraps another

Andrew Left, short seller who campaigned against Valeant, is accused of fraud by SEC

European drug regulator rejects Alzheimer's drug Leqembi, citing safety concerns

Critical Junctures in Gene Therapy Clinical Material Manufacturing: Where Do I Start? And Will It Work?