Com­pound­ing drugs dur­ing the pan­dem­ic: FDA of­fers pol­i­cy clar­i­fi­ca­tions

In re­sponse to “many emails from stake­hold­ers,” the FDA late Mon­day clar­i­fied a few poli­cies re­lat­ed to drug com­pound­ing, in­clud­ing one on the lim­it on the in­ter­state dis­tri­b­u­tion of com­pound­ed drug prod­ucts for phar­ma­cy com­pounders.

Over­all, FDA said its poli­cies are meant to pro­tect pa­tients from un­safe, in­ef­fec­tive and poor-qual­i­ty com­pound­ed drugs while pre­serv­ing ac­cess for pa­tients.

In one of the up­dates, FDA clar­i­fies that a draft guid­ance has not tak­en ef­fect and will be re­vised. The guid­ance notes that FDA will not take ac­tion against cer­tain en­ti­ties as long as drugs are dis­trib­uted on­ly to health­care fa­cil­i­ties that are owned and con­trolled by the same en­ti­ty that owns and con­trols the hos­pi­tal phar­ma­cy and that are lo­cat­ed with­in a one-mile ra­dius of the com­pound­ing phar­ma­cy.

An­oth­er up­date says, “Al­though fed­er­al law spec­i­fies a 5 per­cent lim­it on in­ter­state dis­tri­b­u­tion of com­pound­ed drug prod­ucts for phar­ma­cy com­pounders, we do not in­tend to en­force the 5 per­cent lim­it un­til af­ter the agency has fi­nal­ized a Mem­o­ran­dum of Un­der­stand­ing (MOU) and giv­en states an op­por­tu­ni­ty to sign it. The MOU is cur­rent­ly in draft form.”

FDA al­so clar­i­fies guid­ance from 2018 that says that com­pounders may com­pound drugs on FDA’s short­age list or if they have been dis­con­tin­ued or are no longer mar­ket­ed.

“The agency al­so does not con­sid­er a com­pound­ed drug pro­duced by an out­sourc­ing fa­cil­i­ty as ‘es­sen­tial­ly a copy’ if it is iden­ti­cal or near­ly iden­ti­cal to an FDA-ap­proved drug that is on FDA’s drug short­age list. The agency al­so does not in­tend to take ac­tion un­der this pro­vi­sion if the fa­cil­i­ty fills or­ders for a com­pound­ed drug that is es­sen­tial­ly a copy of an ap­proved drug that has been dis­con­tin­ued and is no longer mar­ket­ed,” FDA said.

In an­oth­er new Q&A on man­u­fac­tur­ing and the sup­ply chain, FDA ex­plains to po­ten­tial ap­pli­cants: “Dur­ing this in­ter­im pe­ri­od, we are uti­liz­ing ad­di­tion­al tools to de­ter­mine the need for an on-site in­spec­tion to sup­port the ap­pli­ca­tion as­sess­ment such as re­view­ing a firm’s pre­vi­ous com­pli­ance his­to­ry, us­ing in­for­ma­tion shar­ing from for­eign gov­ern­ments as part of mu­tu­al recog­ni­tion and con­fi­den­tial­i­ty agree­ments, and re­quest­ing records ‘in ad­vance of or in lieu of’ on-site drug in­spec­tions. If the fi­nal de­ter­mi­na­tion is an on-site in­spec­tion is nec­es­sary, an ap­pli­ca­tion may re­ceive a Com­plete Re­sponse.”

In ad­di­tion, FDA said it’s work­ing to ad­dress the Covid-19 pan­dem­ic by fa­cil­i­tat­ing im­ports of drugs to po­ten­tial­ly treat Covid-19.

Coro­n­avirus (COVID-19) | Drugs


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Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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The flu virus (CDC)

Roche tacks on an­oth­er Xofluza in­di­ca­tion as flu sea­son meets pan­dem­ic

Xofluza was heralded as the first new flu drug in 20 years when it got the FDA OK back in 2018. But even so, Roche saw tough competition from cheaper Tamiflu generics that appeared to be nearly as — if not just as — effective.

Now, the pharma says the drug also can be used to prevent influenza after exposure, snagging a new approval and adding to Xofluza’s appeal as flu season meets the pandemic.

Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.