Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aro­mat­ic amino acid de­car­boxy­lase (AADC) de­fi­cien­cy is an ul­tra-rare ge­net­ic dis­ease that leaves pa­tients un­able to pro­duce cer­tain hor­mones in the brain, such as dopamine and sero­tonin, usu­al­ly lead­ing to de­vel­op­men­tal de­lays, weak mus­cle tone and in­abil­i­ty to con­trol the move­ment of the limbs. It can al­so lead to mul­ti­ple or­gan fail­ure.

To date, there have been no treat­ments ap­proved for AADC de­fi­cien­cy, which has been iden­ti­fied in less than 150 pa­tients.

But now, New Jer­sey biotech PTC Ther­a­peu­tics is one step clos­er to get­ting its gene ther­a­py ap­proved in the EU. The Eu­ro­pean Med­i­cines Agency an­nounced ear­li­er to­day that it would rec­om­mend grant­i­ng a mar­ket­ing au­tho­riza­tion for the one-time gene ther­a­py Up­staza, giv­ing it the “ex­cep­tion­al cir­cum­stances” moniker.

That moniker is giv­en to a type of mar­ket­ing au­tho­riza­tion grant­ed to med­i­cines where the ap­pli­cant is “un­able to pro­vide com­pre­hen­sive da­ta on the ef­fi­ca­cy and safe­ty un­der nor­mal con­di­tions of use, be­cause the con­di­tion to be treat­ed is rare or be­cause col­lec­tion of full in­for­ma­tion is not pos­si­ble or is un­eth­i­cal,” ac­cord­ing to the EMA.

Da­ta come from three tri­als that had en­rolled a com­bined 28 chil­dren. The main fa­vor­able ef­fects at­tained by the par­tic­i­pants in the study were head con­trol and the abil­i­ty to sit unas­sist­ed, the EMA said, adding that con­sul­tants agreed that mean­ing­ful ef­fi­ca­cy was demon­strat­ed in the tri­als

The agency’s CHMP com­mit­tee al­so has asked PTC to sub­mit da­ta show­ing both long-term ef­fi­ca­cy and safe­ty in pa­tients en­rolled in clin­i­cal tri­als based on a 10-year fol­low up and a glob­al reg­istry-based safe­ty study. In the mean­time, an­oth­er EMA com­mit­tee, the Com­mit­tee for Ad­vanced Ther­a­pies, said that the ben­e­fits of Up­staza out­weigh the pos­si­ble risks in pa­tients with AADC de­fi­cien­cy. The most com­mon side ef­fects were el­e­vat­ed body tem­per­a­ture and in­vol­un­tary, er­rat­ic move­ments, known as dysk­i­ne­sia.

Af­ter an au­tho­riza­tion is grant­ed — PTC said in a state­ment it was ex­pect­ing a de­ci­sion in ap­prox­i­mate­ly two months — the next big ques­tion that EU mem­ber states and PTC will face is the price. Gene ther­a­pies, es­pe­cial­ly ones pro­mot­ed to be a one-treat­ment cure, have shown to be ex­treme­ly cost­ly. No­var­tis’s Zol­gens­ma, a gene ther­a­py for a spe­cif­ic type of spinal mus­cu­lar at­ro­phy, owns the du­bi­ous dis­tinc­tion of most ex­pen­sive drug in the world, cost­ing be­tween $1.8 mil­lion and $2.1 mil­lion per pa­tient.

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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

FDA's VRB­PAC votes in fa­vor of adapt­ing the Covid-19 vac­cine to the lat­est Omi­cron vari­ant

The FDA’s Vaccine and Related Biological Products Advisory Committee on Tuesday gave the thumbs up — by a vote of 19-2 — that the FDA should require an Omicron-related component in this next season’s booster dose for Covid-19, which both Pfizer/BioNTech and Moderna are hard at work on.

And while neither booster will likely be ready to go with adequate supplies for all American adults by the beginning of the next school year, the situation is still complex and fluid, with CBER Director Peter Marks telling the committee that it’ll take companies at least three months to ready their supplies for this expected next wave.

Bob Nelsen (Lyell)

UP­DAT­ED: As bear mar­ket con­tin­ues to beat down biotech, ARCH clos­es a $3B ear­ly-stage fund

One of the biggest names in biotech investing has a whole lot of new money to spend.

ARCH Venture Partners closed its 12th venture fund early Wednesday morning, the firm said, bringing in almost $3 billion to invest in early-stage biotechs. The move comes about a year and a half after ARCH announced its previous fund, for almost $2 billion back in January 2021.

In an interview with Endpoints News, ARCH managing director and co-founder Bob Nelsen brushed off concerns about the broader market troubles, saying the downturn that’s seen several biotechs downsize and the XBI fall back to almost pre-pandemic levels won’t impact ARCH’s mindset too much because it typically focuses on company formation.

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Hank Safferstein, Generian CEO

Astel­las sub­sidiary to part­ner with Pitts­burgh up­start in search for 'un­drug­gable' pro­teins

As Astellas continues its drive to build out its gene therapy portfolio and capabilities, a subsidiary of the Japanese pharma company has entered into a collaboration with a little-known Pittsburgh biotech.

Astellas-owned Mitobridge and Generian Pharmaceuticals announced on Wednesday that they will work together in a new deal for “undruggable” protein targets. Generian will net an undisclosed upfront payment and could get up to $180 million in milestones, should anything from its platform prove successful, as well as single-digit royalties on global net sales.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Adam Simpson, Icosavax CEO

Reel­ing from Covid flop, Icosavax says its RSV can­di­date passed ear­ly test. But in­vestors need some more con­vinc­ing

Three months separated from a disappointing readout of its Covid-19 vaccine, Icosavax is back with what it calls positive topline data for a different VLP vaccine candidate — although investors aren’t impressed.

IVX-121, a vaccine candidate for respiratory syncytial virus (RSV), appeared to generate “robust” immune responses among both young and older adults, as measured by neutralizing antibodies, and appeared generally well-tolerated, Icosavax reported.

Sanofi to cut in­sulin prices for unin­sured from $99 to $35, match­ing the in­sulin cap com­ing through Con­gress

As the House-passed bill to cap the monthly price of insulin at $35 nationwide makes its way for a Senate vote soon, Sanofi announced Wednesday morning that beginning next month it will cut the monthly price of its insulins for uninsured Americans to $35, down from $99 previously.

The announcement from Sanofi, which allows the uninsured to buy one or multiple Sanofi insulins (Lantus, Insulin Glargine U-100, Toujeo, Admelog, and Apidra) at $35 for a 30-day supply effective July 1, follows House passage (232-193) of the monthly cap in March, with just 12 Republicans voting in favor of the measure.