Regulatory channel feed

Richard Pops, Alkermes CEO (Patrick T. Fallon/Bloomberg via Getty Images)

Alk­er­mes is hand­ed a CRL for ‘3831, mark­ing an­oth­er set­back on the R&D front

Alkermes $ALKS is going to have to wait some more before launching its anti-psychotic combo drug ALKS-3831. The FDA has handed the biotech a CRL, rejecting the application in the company’s latest setback in R&D.

After getting a majority of the outside experts that reviewed the drug for the FDA to provide a thumbs up on approval, the biotech reported today that their manufacturing operations didn’t pass muster during their remote inspection of records — an alternative to a formal survey the agency adopted last summer as a result of the pandemic.

Pen­ny stock play­er Adamis gets an­oth­er CRL for high-dose nalox­one as shares crater

The FDA has once again shut the door on micro cap biotech Adamis Pharmaceuticals’ high dose naloxone injection.

Regulators handed down the program’s second CRL in nearly 12 months, Adamis announced Monday, sending the penny stock spiraling. The new rejection came as a result of new chemistry, manufacturing and controls issues, though Adamis noted that none of the problems stemmed from the “extractables and leachables testing” problems that caused the first thumbs down.

Cedric Francois, Apellis CEO (Apellis)

Apel­lis' Soliris ri­val gets a date at the FDA for a rare blood dis­or­der, with po­ten­tial­ly $400M worth of im­pli­ca­tions

Apellis $APLS is one step closer to catching its rival for the rare blood disorder PNH.

The Waltham, MA-based biotech announced that the FDA has begun priority review for its experimental medicine pegcetacoplan after the drug beat Alexion’s blockbuster Soliris in a head-to-head Phase III. Regulators have set a PDUFA date for May 14, 2021, and Apellis noted that no adcomm is currently expected.

Investors seemed largely indifferent at the news, with Apellis down by about 0.8% at the end of day Monday. Alexion $ALXN shares, meanwhile, were flat.

Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

Bris­tol My­er­s' $9 Cel­gene CVR is tee­ter­ing on the brink of dis­as­ter as the FDA de­lays the liso-cel ap­pli­ca­tion yet again

The CVR ($BMYRT) tied to Bristol Myers Squibb’s big Celgene buyout took one more step right to the brink Monday afternoon.

As some analysts had fretted about, the FDA was unable to schedule an inspection of one of the manufacturing sites that was to be used for the production of liso-cel, their CAR-T picked up in the buyout, which was originally developed by Juno. As a result, Bristol Myers reported today that the FDA will not meet its PDUFA date today.

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Sanofi grap­ples with re­jec­tion of po­ten­tial first-in-class or­phan drug, faults third-par­ty man­u­fac­tur­ing

A snap review didn’t lead to a quick OK for Sanofi’s sutimlimab. A day after the FDA’s supposed decision date on the orphan drug, the French pharma giant said it was handed a rejection.

The complete response letter cites “certain deficiencies identified by the agency during a pre-license inspection of a third-party facility responsible for manufacturing,” Sanofi wrote. Regulators apparently didn’t take any issue with the clinical data or safety.

Mer­ck­'s Keytru­da nets an­oth­er ap­proval, this time in triple neg­a­tive breast can­cer. Can it catch up to Tecen­triq?

Another day, another win for Merck’s blockbuster Keytruda.

The FDA has granted accelerated approval for the cash cow combined with chemotherapy in triple negative breast cancer, giving the drug the green light in its 18th different cancer. Monday’s new indication comes for patients with PD-L1-expressing tumors with a Combined Positive Score of at least 10.

Merck noted that due to the nature of the accelerated approval, the thumbs up is contingent upon confirmatory trials.

Jean Jacques Bienaimé, BioMarin via YouTube

Bio­Marin CEO Bi­en­aimé beats a re­treat from Eu­rope, yank­ing their ap­pli­ca­tion for he­mo­phil­ia A gene ther­a­py

There will be no near-term OK for BioMarin’s hemophilia A gene therapy valrox in Europe.

Already sent back by the FDA to gather more data on durability after researchers flagged a waning response over several years, the EMA had also pinned a request for extended results — though they were willing to take less than the US agency.

The EMA posted a November 4 letter from BioMarin $BMRN saying they were pulling their application — for now — because they wouldn’t have the added data within the time allowed for it.

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Janet Woodcock, director of the Center for Drug Evaluation and Research (Tom Williams/CQ Roll Call via AP Images)

CDER's Janet Wood­cock: Phar­ma, reg­u­la­tors have lessons to learn from Covid-19

When pharmaceutical companies, health care leaders and regulators finally get a chance to debrief and pull lessons learned from the coronavirus pandemic, a chief takeaway may revolve around the many missed research opportunities. However, learnings from Covid-19 may one day lead to novel trial design and increased cooperation among these players promise a better path forward when speed and safety need equal consideration. This message was among many shared by a leading regulator in a 9 November audio interview with editors at the New England Journal of Medicine (NEJM).

French health prod­ucts agency yanked in­to le­gal bat­tle over birth de­fects caused by Sanofi's De­pakine

A probe into Sanofi’s epilepsy drug Depakine has now entangled France’s National Medicines and Health Products Agency (ANSM), which was indicted on Wednesday for “injuries and manslaughter by negligence” related to birth defects and stillborn births caused by the drug.

Sanofi found itself at the center of a manslaughter investigation in August, several months after it was formally charged. The drug, sodium valproate, has been on the market since 1967 for epilepsy and bipolar disorder. It’s on the WHO’s list of “essential medicines,” and is prescribed in over 100 countries. But according to Reuters, France’s social affairs inspection agency estimates 425 to 450 babies exposed to the drug suffered congenital birth defects or were stillborn from 2006 to 2014.

Ex­pect­ed mis­takes, the need for speed, and change to come: CBER's Pe­ter Marks looks ahead

In a wide-ranging Nov. 6 discussion with the president and CEO of the Friends of Cancer Research, Jeff Allen, CBER director Marks spoke to tempering public expectation, sustaining CBER staff despite a crushing workload during the coronavirus pandemic, and the possibility of more changes in 2021.

Under a new administration, said Marks, speaking at a virtual session of BioPharma Congress 2020, much could change. “So my hope is that we’ve already started the foundation for what needs to happen here.