FDA rolls out an ear­ly and his­toric OK for Spark's pi­o­neer­ing gene ther­a­py -- now let's talk price

Spark Ther­a­peu­tics CSO Kather­ine High and CEO Jeff Mar­raz­zo. Spark


Spark Ther­a­peu­tics $ONCE  has scored an his­toric FDA ap­proval of Lux­tur­na, the world’s first such AAV-de­liv­ered gene ther­a­py de­signed to cure a rare eye dis­ease trig­gered by a ge­net­ic mu­ta­tion.

The drug is OK’d for RPE65 mu­ta­tion linked reti­nal dy­s­tro­phy. The treat­ment us­es a vi­ral vec­tor to in­sert the cor­rect copy of a gene reti­nal cells need to cre­ate a pro­tein that turns light in­to elec­tric sig­nals which can re­store vi­sion lost to the dis­ease.

Scott Got­tlieb

As with the ear­li­er pi­o­neer­ing ap­proval of the world’s first CAR-T, FDA com­mis­sion­er Scott Got­tlieb did the hon­ors in rec­og­niz­ing the im­por­tance of this ap­proval. And he says the agency will make sure that the reg­u­la­to­ry path is straight and clear for the rest of the field look­ing to fol­low­ing Spark’s foot­steps.

“We’re at a turn­ing point when it comes to this nov­el form of ther­a­py and at the FDA, we’re fo­cused on es­tab­lish­ing the right pol­i­cy frame­work to cap­i­tal­ize on this sci­en­tif­ic open­ing,” Got­tlieb not­ed. “Next year, we’ll be­gin is­su­ing a suite of dis­ease-spe­cif­ic guid­ance doc­u­ments on the de­vel­op­ment of spe­cif­ic gene ther­a­py prod­ucts to lay out mod­ern and more ef­fi­cient pa­ra­me­ters — in­clud­ing new clin­i­cal mea­sures — for the eval­u­a­tion and re­view of gene ther­a­py for dif­fer­ent high-pri­or­i­ty dis­eases where the plat­form is be­ing tar­get­ed.”

“This one-time gene ther­a­py for an in­her­it­ed dis­ease rep­re­sents a first-of-its-kind break­through that may lay the ground­work for the de­vel­op­ment of gene ther­a­pies for oth­er con­di­tions that are not ad­e­quate­ly ad­dressed to­day,” said Jef­frey Mar­raz­zo, CEO at Spark Ther­a­peu­tics, in a state­ment. “We of­fer our sin­cere grat­i­tude to the pa­tients and their fam­i­lies as well as the ex­pert in­ves­ti­ga­tors who con­tin­ue to par­tic­i­pate in Lux­tur­na’s clin­i­cal de­vel­op­ment pro­gram.”

The next big step in this process? Mar­raz­zo can tell us how much it will cost. The biotech has been hint­ing that the tick­et will come in at about $1 mil­lion, stir­ring a long run­ning de­bate over a new kind of drug de­signed to last a life­time, but proven to work for on­ly a lim­it­ed amount of time.

Peak sales es­ti­mates tend to hov­er around the $500 mil­lion a year mark.

A Spark spokesper­son said the com­pa­ny wouldn’t re­lease the drug’s price un­til Jan­u­ary, but that Lux­tur­na is ex­pect­ed to be avail­able in se­lect treat­ment cen­ters in Q1 2018.

Spark al­so has a close­ly watched he­mo­phil­ia B gene ther­a­py in the clin­ic, though its mixed da­ta from their he­mo­phil­ia A pro­gram un­der­scored how many hur­dles are left for the lead de­vel­op­ers in the field.

The ap­proval marks yet an­oth­er quick de­ci­sion for the FDA, which had a PDU­FA date for this treat­ment in mid-Jan­u­ary. The agency has been rolling out new drugs this year at a fast pace, look­ing to sur­pass 2015, when 45 new drugs were ap­proved. And the agency seems de­ter­mined to hit that goal post, if not surge past.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

On a roll, Mer­ck blazes through a new seg­ment of the bio­mark­er trail

Merck has notched an approval for using Keytruda to treat a biomarker-based subset of first-line colorectal cancer patients with unresectable or metastatic tumors, as the pharma giant continues to find new niches for its blockbuster PD-1 star.

The OK is significant in a number of ways. Not only does it build on an accelerated approval for all tumors characterized as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR); it also marks the first single treatment for colorectal cancer that doesn’t contain chemotherapy.

Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)

No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.