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.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,700+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,700+ biopharma pros reading Endpoints daily — and it's free.

FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources. 

As­traZeneca's di­a­betes drug Farx­i­ga helps pa­tients with heart dis­ease and with­out di­a­betes in land­mark tri­al

Months ago, data on J&J’s $JNJ Invokana indicated the diabetes drug conferred cardiovascular (CV) benefit in patients who do and do not have preexisting CV disease. On Tuesday, AstraZeneca’s $AZN rival treatment, Farxiga, was shown to cut the risk of CV death or the worsening of heart failure in patients with heart disease, in a landmark trial.

The treatments, in addition to Jardiance from Eli Lilly $LLY, belong to a class of diabetes drugs called sodium-glucose co-transporter 2 (SGLT2) inhibitors, which work by curbing the absorption of glucose via the kidneys so that surplus glucose is excreted through urination.

Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,700+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
Endpoints News

Basic subscription required

Unlock this story instantly and join 57,700+ biopharma pros reading Endpoints daily — and it's free.