Frank Pallone (Kevin Dietsch/Pool via AP Images)

House com­mit­tee seeks more from FDA on in­spec­tion back­log, when to restart work on for­eign sites

House En­er­gy & Com­merce com­mit­tee lead­ers are rais­ing fresh ques­tions about the FDA’s abil­i­ty to con­duct for­eign man­u­fac­tur­ing site in­spec­tions and bring down its grow­ing back­log.

“While we un­der­stand that the emer­gence of COVID-19 re­quired the agency to sus­pend in-per­son in­spec­tion ac­tiv­i­ties tem­porar­i­ly, we re­main con­cerned that more than one year in­to the pan­dem­ic, the strat­e­gy for re­sum­ing all in­spec­tions and ad­dress­ing the back­log of de­layed in­spec­tions re­mains un­clear,” E&C chair Frank Pal­lone (D-NJ) and a group of five oth­er bi­par­ti­san lead­ers of the com­mit­tee wrote to FDA act­ing com­mis­sion­er Janet Wood­cock.

The let­ter fol­lows Wood­cock’s an­nounce­ment last Mon­day that, for the first time since March 2020, the FDA’s in­spec­tions of US-based bio­phar­ma sites have re­turned to nor­mal.

As Wood­cock struck a more op­ti­mistic tone in that an­nounce­ment, the House rep­re­sen­ta­tives note that for­eign fa­cil­i­ties in­spect­ed by US-based staff are on­ly sub­ject to mis­sion-crit­i­cal in­spec­tions, and while the agency has al­ter­na­tive meth­ods to in­spect sites re­mote­ly, “it is large­ly un­clear for which types of in­spec­tions, if any, FDA can ap­pro­pri­ate­ly use these al­ter­na­tives as a full sub­sti­tute for an in-per­son in­spec­tion,” the rep­re­sen­ta­tives wrote.

For ex­am­ple, re­mote eval­u­a­tions can­not sat­is­fy the statu­to­ry re­quire­ment for sur­veil­lance in­spec­tions, as those must be ei­ther on­site or con­duct­ed by the FDA’s for­eign coun­ter­parts. The agency said it post­poned near­ly 8,000 non-mis­sion-crit­i­cal sur­veil­lance in­spec­tions in 2020 due to the pan­dem­ic.

“We are al­so con­cerned that we have not yet seen the full im­pact of de­layed in­spec­tions, par­tic­u­lar­ly in the case of preap­proval in­spec­tions (PAIs). PAIs may oc­cur well in ad­vance of tar­get­ed dates for drug ap­proval de­ci­sions. There­fore, drug ap­pli­ca­tions and ap­provals may be af­fect­ed in the fu­ture if in­spec­tions con­tin­ue to be post­poned, pos­si­bly de­lay­ing pa­tient ac­cess to need­ed med­ica­tions,” the let­ter says.

Ac­cord­ing to a re­port from the FDA in May, an es­ti­mat­ed 68 ap­pli­ca­tions (in­clud­ing 48 for dif­fer­ent drugs) have al­ready been de­layed due to the FDA’s in­abil­i­ty to con­duct pre-ap­proval, pre-mar­ket, or pre-li­cense in­spec­tions as of March.

The law­mak­ers al­so ques­tioned Wood­cock, seek­ing re­spons­es by Aug. 5 on whether the agency has an analy­sis to de­ter­mine how de­layed in­spec­tions may im­pact the agency’s abil­i­ty to make drug ap­proval de­ci­sions in the com­ing months and years.

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Merck CEO Rob Davis

Mer­ck emerges as lead bid­der in po­ten­tial Ac­celeron buy­out with deal pos­si­ble this week — re­port

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Alexander Lefterov/Endpoints News

The coro­n­avirus vac­cine that the world for­got could still help save it

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

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Habib Dable, Acceleron CEO

Days of heat­ed ru­mors cul­mi­nate in a re­port that Ac­celeron is in ad­vanced buy­out talks

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

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Peter Marks (Jim Lo Scalzo/Pool via AP Images)

Tur­moil at CBER: Pe­ter Marks grabs con­trol of FDA's Of­fice of Vac­cines ahead of 2 key ca­reer leader de­par­tures

FDA’s top vaccine official Peter Marks is pulling the plug on a months-long transition for two top career vaccine officials who abruptly called it quits in late August.

Marion Gruber, director of the FDA’s Office of Vaccines Research & Review and 32-year veteran of the agency, her deputy director Phil Krause, announced their departures and then raised concerns with Covid-19 booster shots ahead of and during a recent Covid-19 booster vaccine advisory committee.

Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

Seiz­ing Aduhelm prece­dent, Bio­gen, Ei­sai get the ball rolling on an­oth­er ac­cel­er­at­ed Alzheimer's ap­proval

Biogen — together with partners at Eisai — will be the first to breeze down that Alzheimer’s trail it blazed with the FDA’s historic and controversial accelerated approval of Aduhelm.

Eisai disclosed it’s initiated a rolling BLA submission for lecanemab, or BAN2401, the anti-amyloid beta antibody that Biogen has long touted as a follow-on to Aduhelm (then aducanumab). Following the precedent that the agency has now set, the two companies are gunning for an OK based on data suggesting that the drug could lower amyloid beta plaques.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

New ARIA cas­es dog Bio­gen's Aduhelm launch in an­oth­er po­ten­tial blow to block­buster hopes — an­a­lyst

Perhaps anticipating a walkover with an FDA approval in hand, Biogen has instead faced a wall of pushback over its controversial Alzheimer’s med Aduhelm. With payers and clinics largely staying away, Biogen’s drug is posting some troubling safety signals that could derail the launch even further.

In a note to clients Friday, analysts from Baird revealed new safety data from the FDA’s Adverse Event Reporting System (FAERS) showing a troubling recurrence of ARIA in a crop of patients who have been dosed with Aduhelm.

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Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

Covid-19 roundup: Pfiz­er/BioN­Tech sub­mit vac­cine da­ta to FDA for younger chil­dren; Doc­tors kept pre­scrib­ing hy­drox­y­chloro­quine

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.