Rachelle Jacques, Enzyvant CEO

An FDA mis­take sig­nals like­ly ap­proval for first ther­a­py for chil­dren born with­out a thy­mus

The FDA on Thurs­day ac­ci­den­tal­ly pub­lished a no­tice an­nounc­ing the award of a pri­or­i­ty re­view vouch­er to rare dis­ease drug de­vel­op­er En­zy­vant for its new re­gen­er­a­tive ther­a­py for the treat­ment of pe­di­atric pa­tients with con­gen­i­tal athymia.

The on­ly prob­lem? The treat­ment still hasn’t won FDA ap­proval. The agency told End­points the no­tice was pub­lished in er­ror and will be with­drawn. The re­lease of the PRV no­tice, which typ­i­cal­ly come days or weeks af­ter an ap­proval is an­nounced, puz­zled the com­pa­ny.

Vivek Ra­maswamy’s En­zy­vant, sold as part of a $3 bil­lion deal with Sum­it­o­mo Dainip­pon Phar­ma, said in an emailed state­ment that it “has not re­ceived ap­proval no­ti­fi­ca­tion from the FDA. Our PDU­FA date re­mains Oc­to­ber 8. En­zy­vant found out about the PRV when it was pub­lished in the Fed­er­al Reg­is­ter and is work­ing with the agency to de­ter­mine what this means.”

The treat­ment in ques­tion, po­ten­tial­ly to be known as Rethymic (al­lo­gene­ic processed thy­mus tis­sue-agdc), would be the first for the ul­tra-rare con­di­tion in which chil­dren are born with­out a thy­mus, known as con­gen­i­tal athymia, which can lead to pro­found im­mun­od­e­fi­cien­cy is­sues and make them high­ly sus­cep­ti­ble to in­fec­tions. Ac­cord­ing to En­zy­vant, pa­tients with con­gen­i­tal athymia, usu­al­ly about 20 per year in the US, die from in­fec­tions or au­toim­mune man­i­fes­ta­tions by age two or three.

This has been a long road to ap­proval (if an ap­proval oc­curs) as En­zy­vant re­ceived a CRL for the treat­ment in De­cem­ber 2019 due to sev­er­al reg­u­la­to­ry re­quests re­lat­ed to chem­istry, man­u­fac­tur­ing and con­trols.

En­zy­vant CEO Rachelle Jacques told End­points News in April that it re­solved is­sues re­lat­ed to the CRL:

We did need to do some fa­cil­i­ties con­struc­tion and we did that dur­ing Covid. That’s some­thing that we didn’t an­tic­i­pate im­me­di­ate­ly when we had the let­ter in our hands, but cer­tain­ly as we talked with the agency that was very clear that was a so­lu­tion. A lot of the oth­er work was re­al­ly just doc­u­ment­ing at a very gran­u­lar lev­el some of the process­es and so on as well as some ad­di­tion­al stud­ies we did to sup­ple­ment the da­ta we pro­vid­ed on the over­all man­u­fac­tur­ing process — and those were long lead time items as well.

Ed­i­tor’s note: Ar­ti­cle up­dat­ed with com­ment from FDA. 

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

In­cyte yanks EU ap­pli­ca­tion for PD-1 drug, cit­ing in­abil­i­ty to an­swer reg­u­la­tors' 'ma­jor con­cern­s'

Hoping to wedge its way into a crowded PD-(L)1 field, Incyte faced a huge setback when the FDA sent back its application for an initial indication earlier this year. Now, the drugmaker is giving up hope in Europe as well.

Incyte has withdrawn its EU application for PD-1 drug Zynyz, or retifanlimab, after saying it couldn’t adequately address the CHMP’s questions over survival data underpinning its market hopes in squamous cell carcinoma of the anal cavity, the EMA revealed.

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Jacob Van Naarden, Senior VP, CEO of Loxo Oncology at Lilly; President, Lilly Oncology

Eli Lil­ly bags FDA nod for Verzenio in ear­ly breast can­cer, but a con­tro­ver­sial di­ag­nos­tic could dog its roll­out

As Eli Lilly works to consolidate its internal and Loxo teams into an oncology powerhouse, the drug giant is putting high hopes on CDK 4/6 inhibitor Verzenio to help drive the portfolio into the future. Now, the drug has scored a paradigm-altering win in early breast cancer — but will a controversial companion diagnostic hamstring Lilly’s market plans?

The FDA on Wednesday approved CDK 4/6 inhibitor Verzenio in combination with physician’s-choice endocrine therapy to cut the risk of relapse in patients with high-risk HR-positive, HER2-negative breast cancer, Lilly said in a release.

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