FDA ap­proves 5 new cost­ly drugs well ahead of PDU­FA dates

Since 21 Oc­to­ber, the FDA has been on a tear in ap­prov­ing five new drugs (all with list prices of more than $100,000 per year) months ahead of when they were ex­pect­ed to be ap­proved.

For in­stance, the FDA signed off on Ver­tex Phar­ma­ceu­ti­cals’ Trikaf­ta (elex­a­caftor/iva­caftor/teza­caftor), a new treat­ment for those with the most com­mon cys­tic fi­bro­sis mu­ta­tion, af­ter on­ly three months of re­view and well ahead of its 19 March 2020 user fee ac­tion date.

On 14 No­vem­ber, more than three months ahead of its 27 Feb­ru­ary 2020 ac­tion date, the FDA grant­ed ac­cel­er­at­ed ap­proval to BeiGene’s Brukin­sa (zanubru­ti­nib) for the treat­ment of pa­tients with man­tle cell lym­phoma who have re­ceived at least one pri­or ther­a­py.

One day lat­er, No­var­tis’ Adakveo (crizan­l­izum­ab-tm­ca) won ap­proval for its sick­le cell dis­ease treat­ment two months ahead of its PDU­FA date in mid-Jan­u­ary 2020. And yes­ter­day, the FDA grant­ed an ac­cel­er­at­ed ap­proval to an­oth­er sick­le cell drug, Glob­al Blood Ther­a­peu­tics’ Oxbry­ta (vox­elo­tor), three months ahead of its PDU­FA date.

Al­ny­lam Phar­ma­ceu­ti­cals’ Givlaari (givosir­an), mean­while, had a PDU­FA date of 4 Feb­ru­ary 2020, but won ap­proval on 20 No­vem­ber. But oth­er re­cent ap­provals, like SK Life Sci­ences’ Xco­pri (cenoba­mate tablets) to treat par­tial-on­set seizures in adults, and Sh­iono­gi’s com­pli­cat­ed uri­nary tract in­fec­tion drug Fetro­ja (ce­fide­ro­col), won ap­provals near their PDU­FA dates.

The string of quick ap­provals may pro­vide more am­mu­ni­tion for those who crit­i­cize the agency for mov­ing too quick­ly. An ar­ti­cle in JA­MA In­ter­nal Med­i­cine last sum­mer found that few can­cer drugs ap­proved via the ac­cel­er­at­ed ap­proval path­way im­proved sur­vival in con­fir­ma­to­ry tri­als.

How­ev­er, view­ers of the re­cent Sen­ate com­mit­tee hear­ing con­sid­er­ing a new FDA com­mis­sion­er have seen there are still sen­a­tors who be­lieve the FDA is not mov­ing quick­ly enough with some ap­provals.

As the pro­por­tion of new drugs re­ceiv­ing ex­pe­dit­ed ap­provals in re­cent years has been in­creas­ing, so has the num­ber of ap­provals for rare dis­eases.

Janet Wood­cock FDA

Janet Wood­cock, di­rec­tor of the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search, ex­plained re­cent­ly that the agency is work­ing on its own analy­ses to pro­vide “a more ro­bust re­sponse” to these cri­tiques of its ap­proval stan­dards.

She al­so ex­plained how the high num­ber of ap­provals in re­cent years for rare dis­eases may be in­flu­enc­ing this per­cep­tion of a low­er bar, es­pe­cial­ly as more treat­ments are ap­proved on the ba­sis of a sin­gle-arm study or with an ex­ter­nal con­trol group. In ad­di­tion, she point­ed to the “as­tound­ing­ly” high launch prices for some of these rare dis­ease treat­ments that may al­so be part of the rea­son for the push­back.

In­deed, be­fore dis­counts, Trikaf­ta will cost $311,503 an­nu­al­ly, Brukin­sa will cost $12,935 for a 30-day sup­ply, Adakveo will cost be­tween $7,000 and $9,500 per month ($84,000 to $114,000 per year), Oxbry­ta will cost $125,000 per year and Givlaari will cost $575,000 per year.

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At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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What's fair? New ICER re­port shows pay­ers gen­er­al­ly en­sur­ing fair ac­cess to drugs

The nonprofit Institute for Clinical and Economic Review on Wednesday released a new report highlighting the ways in which payers are generally ensuring fair access to prescription drugs, even when based on a set of criteria set by the nonprofit.

While noting the lack of transparency hindered the report’s results, ICER said that the “great majority” of payer policies in the formularies evaluated are structured in a way to support many key elements of how ICER defines “fair access.”

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

J&J and Sanofi's mul­ti­ple myelo­ma tit-for-tat con­tin­ues, as sub­cu­ta­neous Darza­lex wins com­bo ap­proval

J&J and Sanofi have gone back and forth in their multiple myeloma tug-of-war. Earlier this year, Sanofi notched an approval of Sarclisa in combination with Amgen’s Kyprolis to try to outflank the big conglomerate, but J&J is clapping back.

Wednesday afternoon, Amgen announced that the subcutaneous version of J&J’s blockbuster Darzalex is also now approved as a combo with Kyprolis and dexamethasone. The green light came through for adults with relapsed or refractory multiple myeloma who had progressed on one to three earlier lines of therapy.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.