FDA ap­provals roundup: Zo­genix's Fin­tepla, Ul­tragenyx's Do­jolvi, and Genen­tech's Ph­es­go

A week­ly up­date on new drug ap­provals and in­di­ca­tions from the FDA:

New ap­provals

Fin­tepla gets the go-ahead for Dravet syn­drome

Zo­genix’s Fin­tepla (fen­flu­ramine) has been ap­proved for the treat­ment of seizures as­so­ci­at­ed with Dravet syn­drome in pa­tients aged 2 years or old­er. The syn­drome is a rare, life-threat­en­ing form of epilep­sy.

The ap­proval was based on find­ings from two clin­i­cal stud­ies in 202 par­tic­i­pants aged be­tween 2 and 18 years, in which the change from base­line in fre­quen­cy of con­vul­sive seizures was mea­sured. In both stud­ies, chil­dren who re­ceived Fin­tepla had sig­nif­i­cant­ly greater re­duc­tions in the fre­quen­cy of con­vul­sive seizures, com­pared with those re­ceiv­ing place­bo. The re­duc­tions were seen with­in 3-4 weeks of ther­a­py ini­ti­a­tion and re­mained gen­er­al­ly con­sis­tent dur­ing the treat­ment pe­ri­ods of 14-15 weeks.

Fin­tepla is a Sched­ule IV con­trolled sub­stance, car­ry­ing a boxed warn­ing that it is as­so­ci­at­ed with valvu­lar heart dis­ease and pul­monary ar­te­r­i­al hy­per­ten­sion. It is avail­able on­ly through a re­strict­ed drug dis­tri­b­u­tion pro­gram, un­der a risk eval­u­a­tion and mit­i­ga­tion strat­e­gy (REMS) stip­u­lat­ing that pre­scribers and pa­tients must com­ply with the re­quired car­diac mon­i­tor­ing with echocar­dio­grams to re­ceive the ther­a­py.

The ap­pli­ca­tion was grant­ed pri­or­i­ty re­view. The ther­a­py re­ceived or­phan drug des­ig­na­tion.

Do­jolvi picks up ap­proval for rare ge­net­ic dis­or­der

Ul­tragenyx’s Do­jolvi (tri­hep­tanoin) has been ap­proved for the treat­ment of pe­di­atric and adult pa­tients with mol­e­c­u­lar­ly con­firmed long-chain fat­ty acid ox­i­da­tion dis­or­ders (LC-FAOD).

LC-FAOD are a group of rare, life-threat­en­ing ge­net­ic dis­or­ders in which the body is not able to con­vert long-chain fat­ty acids in­to en­er­gy. Do­jolvi is a source of calo­ries and fat­ty acids for these pa­tients.

Safe­ty da­ta for the ther­a­py came from 79 pa­tients with LC-FAOD who re­ceived Do­jolvi in 2 stud­ies, an open-la­bel, 78-week study with 29 pa­tients (Study 1), fol­lowed by an open-la­bel ex­ten­sion study (Study 2). In all, 24 pa­tients from Study 1 con­tin­ued in­to Study 2. Pa­tients ranged in age from 4 months to 63 years. They re­ceived dai­ly dos­es of Do­jolvi for a mean du­ra­tion of 23 months. The most com­mon ad­verse re­ac­tions to the study drug in­clud­ed ab­dom­i­nal (60%), di­ar­rhea (44%), vom­it­ing (44%), and nau­sea (14%). A third study, a 4-month, dou­ble-blind, ran­dom­ized-con­trolled study re­port­ed ad­verse re­ac­tions sim­i­lar to those from the first two stud­ies.

The agency pre­vi­ous­ly grant­ed the ap­pli­ca­tion rare pe­di­atric dis­ease and fast track des­ig­na­tions. Ul­ti­mate­ly, it as­signed a stan­dard re­view des­ig­na­tion with a Pre­scrip­tion Drug User Fee Act tar­get date of Ju­ly 31.

New in­di­ca­tions

Ph­es­go com­bo OK’d for home ad­min­is­tra­tion for HER2+ breast can­cer

Genen­tech’s Ph­es­go (per­tuzum­ab, trastuzum­ab, and hyaluronidase–zzxf in­jec­tion) has been ap­proved as a ther­a­py for adult pa­tients with metasta­t­ic HER2-pos­i­tive breast can­cer and for adults with ear­ly HER2-pos­i­tive breast can­cer, based on the re­sults of FDA-ap­proved com­pan­ion di­ag­nos­tic test.

Ph­es­go is ini­tial­ly used in com­bi­na­tion with chemother­a­py and could con­tin­ue to be ad­min­is­tered at home by a qual­i­fied health care pro­fes­sion­al once the chemother­a­py reg­i­men is fin­ished.

The drug con­tains a fixed-dose com­bi­na­tion of per­tuzum­ab and trastuzum­ab with hyaluronidase. Its ap­proval was based on the re­sults of a non­in­fe­ri­or­i­ty study in pa­tients with HER2-pos­i­tive ear­ly breast can­cer show­ing that the drug’s ef­fi­ca­cy and safe­ty were com­pa­ra­ble with IV per­tuzum­ab and IV trastuzum­ab. Ad­min­is­tra­tion-re­lat­ed re­ac­tions were high­er with Ph­es­go be­cause of the sub­cu­ta­neous route of ad­min­is­tra­tion.

“As part of the FDA’s on­go­ing com­mit­ment to ad­dress the nov­el coro­n­avirus pan­dem­ic, we con­tin­ue to keep a strong fo­cus on pa­tients with can­cer who con­sti­tute a vul­ner­a­ble pop­u­la­tion at risk of con­tract­ing the dis­ease. At this crit­i­cal time, we con­tin­ue to ex­pe­dite on­col­o­gy prod­uct de­vel­op­ment. This ap­pli­ca­tion was ap­proved about four months ahead of the FDA goal date,” said Richard Paz­dur, di­rec­tor of the agency’s On­col­o­gy Cen­ter of Ex­cel­lence and act­ing di­rec­tor of the Of­fice of On­co­log­ic Dis­eases in the Cen­ter for Drug Eval­u­a­tion and Re­search.

Baven­cio gets new in­di­ca­tion for urothe­lial car­ci­no­ma

EMD Serono/Pfiz­er’s Baven­cio (avelum­ab) has re­ceived an ex­pand­ed in­di­ca­tion for main­te­nance treat­ment of pa­tients with lo­cal­ly ad­vanced or metasta­t­ic urothe­lial car­ci­no­ma (UC) that has not pro­gressed with first-line, plat­inum-con­tain­ing ther­a­py. It is the first ap­proved im­munother­a­py to show sig­nif­i­cant im­prove­ment in over­all sur­vival in the first-line set­ting.

The ap­pli­ca­tion orig­i­nal­ly re­ceived break­through ther­a­py des­ig­na­tion and was grant­ed pri­or­i­ty re­view, which was com­plet­ed un­der the re­al-time on­col­o­gy re­view.

The hu­man an­ti-pro­grammed death lig­and-1 an­ti­body has a pre­vi­ous ap­proval for lo­cal­ly ad­vanced or metasta­t­ic UC that has pro­gressed dur­ing or af­ter plat­inum-con­tain­ing chemother­a­py. It has al­so been ap­proved in com­bi­na­tion with ax­i­tinib for the first-line treat­ment of pa­tients with ad­vanced re­nal cell car­ci­no­ma, and for treat­ing Merkel cell car­ci­no­ma in adults and pe­di­atric pa­tients old­er than 12 years and old­er with metasta­t­ic dis­ease.

Keytru­da ex­pand­ed for in­cur­able CRC, cSCC

Mer­ck’s Keytru­da (pem­brolizum­ab) has re­ceived two ex­pand­ed in­di­ca­tions, one, as a first-line treat­ment for pa­tients with un­re­sectable or metasta­t­ic mi­crosatel­lite in­sta­bil­i­ty-high or mis­match re­pair de­fi­cient col­orec­tal can­cer, and a sec­ond, for treat­ing pa­tients with in­cur­able re­cur­rent or metasta­t­ic cu­ta­neous squa­mous cell car­ci­no­ma (cSCC).

The ap­proval for CRC was grant­ed less than a month af­ter the com­pa­ny sub­mit­ted a sup­ple­men­tal bi­o­log­ics li­cense ap­pli­ca­tion (sBLA) based on re­sults from the KEYNOTE-177 tri­al. It is the first sin­gle-agent, an­ti-PD-1 ther­a­py ap­proved as a first-line ther­a­py for these pa­tients.

The sBLA was re­viewed un­der the agency’s re­al-time on­col­o­gy re­view pi­lot pro­gram. Re­view was al­so done un­der Pro­ject Or­bis, which al­lows for con­cur­rent sub­mis­sion and re­view of on­col­o­gy drugs among in­ter­na­tion­al part­ners. For this ap­pli­ca­tion, the agency is col­lab­o­rat­ing with the Aus­tralian Ther­a­peu­tic Goods Ad­min­is­tra­tion, Health Cana­da and Swissmedic on their on­go­ing re­view of the ap­pli­ca­tion.

The ex­tend­ed ap­proval for cSCC was based on find­ings from the KEYNOTE-629 tri­al.


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Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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Sean Nolan and RA Session II

Less than 3 months af­ter launch, the AveX­is crew’s Taysha rais­es $95M Se­ries B. Is an IPO next?

The old AveXis team is moving quickly in Dallas.

Three months ago, they launched Taysha with $30 million in Series A funding and a pipeline of gene therapies out of UT Southwestern. Now, they’ve announced an oversubscribed $95 million Series B. And the biotech is declining all interview requests on the news, the kind of broad silence that can indicate an IPO is in the pipeline.

Biotechs, including those relatively fresh off launch, have been going public at a frenzy since the pandemic began. Investors have showed a willingness to put upwards of $200 million to companies that have yet to bring a drug into the clinic. Still, if Taysha were to go public in the near future, it would be perhaps the shortest path from launch to IPO in recent biotech memory.

UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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RA, No­var­tis back Gen­tiBio's seed round, plans to launch de­vel­op­ment of En­gTreg ther­a­pies

Boston, MA-based startup GentiBio landed a $20 million seed fund from three investors to dive into engineered regulatory T cell (EngTreg) development.

Marquee investors OrbiMed, Novartis Venture Fund and RA Capital Management have backed GentiBio’s mission to develop EngTregs for the treatment of autoimmune, alloimmune, autoinflammatory, and allergic diseases. Unlike other companies studying treatments using a patient’s own Tregs, GentiBio plans to make use of CD4+ immune cells, found in the blood.

Paul Laikind, ViaCyte CEO

Stem cell play­er Vi­a­Cyte ex­pands col­lab­o­ra­tion with Gore to de­vel­op sub­cu­ta­neous di­a­betes treat­ment

Longtime stem cell player ViaCyte has teamed up with a materials science company in an effort to solve immunosuppression challenges and advance its type 1 diabetes treatments.

Expanding on an existing collaboration, ViaCyte and W.L. Gore have agreed to combine the biotech’s PEC-Encap candidate with a Gore-produced membrane in what they hope will eliminate the need for immunosuppressive drugs. Such treatments have created foreign body responses in the past, and stamping these reactions out is the main goal, ViaCyte CEO Paul Laikind said.

My­ovant lands a fresh $200M loan as FDA mar­ket­ing de­ci­sion looms; Amarin goes it alone in Eu­rope

Myovant is getting ready to roll out its commercial operations to back relugolix, now under FDA review for prostate cancer.

The startup has added a fresh $200 million in support from Sumitomo Dainippon Pharma, which controls a majority of the stock $MYOV. Sumitomo is handing the cash over as a loan, bringing its total to $600 million. Myovant — which is gearing up for a showdown with AbbVie — has also filed an NDA to sell relugolix for uterine fibroids and recently posted positive late-stage data for endometriosis.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Mod­er­na CEO Stéphane Ban­cel out­lines a prospec­tive moth­er­lode of Covid-19 vac­cine rev­enue — will a back­lash fol­low?

Moderna shows no sign of slowing down, or turning charitable when it comes to pricing supplies of its Covid-19 vaccine.

One of the leaders in the Phase III race to get a Covid-19 vaccine across the finish line in record time, Moderna says it’s on track to complete enrollment in one of the most avidly watched studies in the world next month. And the biotech has already banked some $400 million in deposits for vaccine supply as it works through negotiations with countries around the world — as CEO Stéphane Bancel sets out to hire a commercial team.

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J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Covid-19 roundup: J&J and BAR­DA agree to $1 bil­lion for 100 mil­lion dos­es; Plas­ma re­duces mor­tal­i­ty by 50% — re­ports

J&J has become the latest vaccine developer to agree to supply BARDA with doses of their Covid-19 vaccine, signing an agreement that will give the government 100 million doses in exchange for $1 billion in funding.

The agreement, similar to those signed by Novavax, Sanofi and AstraZeneca-Oxford, provides funding not only for individual doses but to help J&J ramp up manufacturing. Pfizer, by contrast, received $1.95 billion for the doses alone. Still, if one looked at each agreement as purchase amounts, J&J’s deal would be $10 per dose, slotting in between Novavax’s $16 per dose and AstraZeneca’s $4 per dose.

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