Regulation

Dec. 1 Quick Takes: broader label for Blueprint; plus Minerva, Biohaven-Sosei, Genetron, PegBio, drug imports

Blueprint gains broader FDA label for Gavreto FDA approved Gavreto pralsetinib from Blueprint Medicines Corp. (NASDA:BLUE) to treat advanced or metastatic RET-mutant medullary thyroid cancer and RET fusion-positive, radioactive iodine-refractory thyroid cancer in patients at...

With Oxlumo’s approval, Alnylam aims to speed access with new value-based pricing framework

With the approval of Oxlumo to treat primary hyperoxaluria, Alnylam is aiming to accelerate patient access to the ultra-rare disease therapy by building on the performance- and prevalence-based pricing...

FDA says WHO study does not refute efficacy of Veklury for COVID-19

FDA on Wednesday made public the review that led to approval of Gilead’s Veklury remdesivir for treatment of hospitalized COVID-19 patients, and explained why it believes the WHO-sponsored SOLIDARITY trial that failed to demonstrate a...

Distribution of Regeneron’s COVID-19 antibody cocktail to begin Tuesday

Starting Tuesday, distribution of Regeneron’s antibody cocktail for COVID-19 will begin via an existing network of hospitals and associated facilities, with plans for a subsequent expansion that will include a broader range of outpatient centers....

Nov. 20 Quick Takes: Approvals in Europe for Alnylam, Alexion therapies; plus FDA priority review for ADC, Sanofi and more

EC approves Alnylam, Alexion therapiesAlnylam Pharmaceuticals Inc. (NASDAQ:ALNY) said the European Commission approved Oxlumo lumasiran, an RNAi therapy targeting the HAO1 mRNA that encodes glycolate oxidase, to treat primary hyperoxaluria in all age groups. The...

WHO counters FDA as it recommends against Veklury for COVID-19

The WHO’s new guidance counters FDA’s October approval of Gilead’s Veklury for hospitalized patients, adding to the debate around the drug’s use and variable performance in randomized, controlled trials. In The BMJ on Thursday, the WHO issued a...

Dec. 10 FDA panel could be gateway to first emergency authorization of COVID-19 vaccine

A Dec. 10 FDA advisory committee meeting could be the springboard for emergency use authorization for BNT162b2, enabling high-risk patients to receive by mid-December the first doses of the mRNA vaccine candidate against SARS-CoV-2 from BioNTech and Pfizer....

Alkermes expects quick resolution to FDA setback for antipsychotic

Alkermes expects a swift resolution to a manufacturing issue that led to FDA’s issuance of a complete response letter for ALKS 3831, but the company isn’t yet committing to a launch timeline for the antipsychotic...

FDA to post data on COVID-19 therapeutic, vaccine EUAs

Responding to public criticism of some of its COVID-19 emergency use authorization decisions, as well as a recommendation from a government watchdog agency, FDA announced Tuesday that it will...

Another U-turn for COVID-19 testing regulation as HHS requires FDA to re-prioritize LDT review

The latest switchback in the U.S.’s regulation of COVID-19 tests sees HHS requiring FDA to review tests it previously said the agency lacked authority over, highlighting the challenge of pursuing a deregulatory agenda in the midst...

BioMarin withdraws MAA for hemophilia gene therapy following safety, durability concerns

Among CHMP’s monthly roundup of opinions was an MAA withdrawal for BioMarin’s hemophilia gene therapy linked to EMA concerns about safety and durability — issues that FDA raised in August. EMA said...

FDA addresses health disparities in final guidance on clinical trial diversity

FDA released on Monday its final guidance on enhancing diversity in clinical trials with recommendations to reduce underrepresentation including adaptive study designs, targeted population enrollment and broadened inclusion criteria. Though the guidance has been in the...

Label expansions coming at a rapid clip for Foundation’s blood-based companion cancer diagnostic

In the past three weeks, FDA has added four companion diagnostic indications to the label of Foundation Medicine’s liquid biopsy test, growing the number of genetic biomarkers indicated for matching patients to targeted therapies. The...

EUA for Lilly, AbCellera COVID mAb despite Phase II miss for authorized dose

FDA’s first emergency use authorization for a SARS-CoV-2-targeting mAb covers the lowest dose of Lilly and AbCellera’s candidate to treat COVID-19 outpatients, a dose that was not effective in the Phase...

Data Byte: FDA’s NME approvals tally closes in on 2019 count

2020 is on track to be another strong year for FDA approvals of new molecular entities, with 42 approved year-to-date. With two months left in the year, the total is already higher than all but...