No­var­tis drums up EU launch plans as CHMP gives thumbs up to Spark's gene ther­a­py Lux­tur­na

Nine months af­ter the FDA gave its land­mark OK for Spark Ther­a­peu­tics’ trail­blaz­ing gene ther­a­py Lux­tur­na, Eu­ro­pean reg­u­la­tors have sig­naled that they are ready to green­light the vi­sion loss treat­ment, too.

Paul Hud­son

The team at Spark $ONCE isn’t the on­ly ones cel­e­brat­ing the pos­i­tive opin­ion from the Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use: No­var­tis, which grabbed ex-US rights to Lux­tur­na ear­li­er this year in a $170 mil­lion deal, will be com­mer­cial­iz­ing the ther­a­py once it’s ap­proved in Eu­rope.

“To­day’s pos­i­tive CHMP opin­ion rep­re­sents a sig­nif­i­cant step in our jour­ney to­ward ad­vanc­ing po­ten­tial­ly life-chang­ing cell and gene ther­a­pies in oph­thal­mol­o­gy,” said Paul Hud­son, CEO of No­var­tis Phar­ma­ceu­ti­cals, in a state­ment. “We look for­ward to work­ing with Spark Ther­a­peu­tics and the EMA to es­tab­lish ac­cess and reimag­ine care for peo­ple in the EU who face the threat of to­tal blind­ness from this in­her­it­ed reti­nal dis­ease.”

Jef­frey Mar­raz­zo

While (un­like in the US) Lux­tur­na won’t be the first gene ther­a­py ap­proved in the EU — Glax­o­SmithK­line and uniQure beat them to it with their ther­a­pies for “bub­ble boy syn­drome” and fa­mil­ial lipopro­tein li­pase de­fi­cien­cy — No­var­tis is hop­ing it would be the first one with a re­al com­mer­cial prospect.

Price would be key here, es­pe­cial­ly as the phar­ma gi­ant faces with a group of sin­gle pay­er op­er­a­tions with con­sid­er­able lever­age over pric­ing.

Some an­a­lysts have al­so ex­pressed con­cerns about the small mar­ket: Lux­tur­na is de­signed as a one-time treat­ment for a rare form of vi­sion loss (which can even­tu­al­ly re­sult in reti­nal blind­ness) caused by mu­ta­tions in the RPE65 mu­ta­tions, which af­fect 1 in 200,000 peo­ple.

In the US, Spark CEO Jeff Mar­raz­zo has painstak­ing­ly laid out a pric­ing mod­el in hopes of con­vinc­ing pay­ers to cov­er the $850,000 pro­ce­dure — $425,000 per eye — for a tiny group of un­der 2,000 po­ten­tial pa­tients, with few­er than 20 new cas­es per year. The biotech booked $6.7 mil­lion in net sales for the first six months of the year, but it’s still too ear­ly to tell how that’s work­ing.

The pos­i­tive CHMP opin­ion is based on a da­ta pack­age that in­clud­ed a to­tal of 43 pa­tients with in­her­it­ed reti­nal dis­ease caused by RPE65 mu­ta­tions. A mar­ket­ing au­tho­riza­tion from the Eu­ro­pean Com­mis­sion is ex­pect­ed to fol­low with­in two months.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

UP­DAT­ED: EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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