June Raine, MHRA chief executive (AP Photo/Frank Augstein, Pool)

UK could al­low rec­i­p­ro­cal drug ap­provals with US FDA as soon as 2024

An in­tel­lec­tu­al prop­er­ty law firm says that re­cent guid­ance from the UK’s Med­i­cines and Health­care prod­ucts Reg­u­la­to­ry Agency hints at the pos­si­bil­i­ty of the agency al­low­ing ap­provals from oth­er reg­u­la­tors, in­clud­ing pos­si­bly the US FDA, to play a role in UK reg­u­la­to­ry process­es.

Marks & Clerk, based out of Lon­don, wrote in a blog post that guid­ance from the UK’s reg­u­la­to­ry agency from late Jan­u­ary is pro­vid­ing more con­text in­to po­ten­tial in­ter­na­tion­al col­lab­o­ra­tion with oth­er reg­u­la­tors. How that would come about is af­ter the UK’s “Re­liance Pro­ce­dure” — a way to en­sure pa­tients in Great Britain post-Brex­it could still ac­cess new med­i­cines — ex­pires.

The pro­ce­dure, in short, al­lows MHRA to uti­lize pos­i­tive EC de­ci­sions to fast-track the ap­proval process for mar­ket­ing au­tho­riza­tions in Great Britain. Back in Sep­tem­ber, the agency an­nounced that the pro­ce­dure would be ex­tend­ed through the end of 2023.

As for what would be next, the MHRA put out word on Jan. 24:

From 1 Jan­u­ary 2024 you can ap­ply to MHRA through our new in­ter­na­tion­al recog­ni­tion frame­work, which will have re­gard to de­ci­sions al­ready made by the Eu­ro­pean Med­i­cines Agency and cer­tain oth­er reg­u­la­tors.

The law firm added at the time that it re­mains to be seen which reg­u­la­tors would be in­clud­ed in the new sys­tem, but it ref­er­enced the sim­i­lar­i­ty of in­ter­na­tion­al col­lab­o­ra­tion akin to Pro­ject Or­bis — a pro­gram set up by the FDA in 2019 to col­lab­o­rate with dif­fer­ent in­ter­na­tion­al agen­cies on sub­mis­sion and re­view of can­cer treat­ments. The project now in­cludes reg­u­la­tors from the US, UK, Aus­tralia and five oth­er au­thor­i­ties.

The firm added that the an­nounce­ment would “no doubt be wel­come news to com­pa­nies who see Great Britain as a key part of a mul­ti-ju­ris­dic­tion­al launch strat­e­gy.” The firm ad­di­tion­al­ly not­ed that al­low­ing the MHRA to es­sen­tial­ly “pig­gy-back” on au­tho­riza­tions from oth­er reg­u­la­tors could have ad­van­tages.

While FDA did not com­ment to End­points News be­fore dead­line, the MHRA on­ly re­it­er­at­ed what it had said be­fore: “We will com­mu­ni­cate who these ad­di­tion­al reg­u­la­tors are and pub­lish de­tailed guid­ance about this new frame­work in due course, in­clud­ing any tran­si­tion arrange­ments for ap­pli­ca­tions re­ceived un­der ex­ist­ing frame­works.”

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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