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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Fireside chat between Hal Barron and John Carroll, UKBIO19

It’s time we talked about bio­phar­ma — live in Lon­don next week

Zoom can only go so far. And I think at this stage, we’ve all tested the limits of staying in touch — virtually. So I’m particularly happy now that we’ve revved up the travel machine to point myself to London for the first time in several years.

Whatever events we have lined up, we’ve always built in plenty of opportunities for all of us to get together and talk. For London, live, I plan to be right out front, meeting with and chatting with the small crowd of biopharma people we are hosting on October 12 at Silicon Valley Bank’s London headquarters. And there’s a lengthy mixer at the end I’m most looking forward to, with several networking openings between sessions.

Pfizer and BioNTech's original Marvel comic book links evolving Covid vaccine science to Avengers' evolving villain-fighting tools.(Source: Pfizer LinkedIn post)

Pfiz­er, BioN­Tech part­ner with Mar­vel for Avengers and Covid-fight­ing com­ic book

Pfizer and BioNTech are collaborating with Marvel to celebrate “everyday” people getting Covid-19 vaccines in a custom comic book.

In the “Everyday Heroes” digital comic book, an evolving Ultron, one of the Avengers’ leading villains, is defeated by Captain America, Ironman and others. The plotline and history of Ultron is explained by a grandfather who is waiting with his family at a clinic for Covid-19 vaccinations.

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Eli Lil­ly and Te­va pre­pare for court bat­tle over mi­graine med ri­val­ry

It looks like Eli Lilly and Teva Pharmaceuticals are going to trial.

A federal appeals court on Monday refused to invalidate three of Teva’s patents for its migraine treatment Ajovy, while also declining to issue a summary judgment in favor of either company, which would effectively end the case without a full trial.

Teva filed suit against Lilly back in 2018, alleging that the company infringed upon nine patents with its rival migraine drug Emgality. The rival drugs were both approved in September 2018 for the preventative treatment of migraine, and are designed to block calcitonin gene-related peptide (CGRP), a protein associated with the onset of migraine pain.

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Tim Miller, Forge Biologics CEO

CNS-fo­cused gene ther­a­py biotech forges man­u­fac­tur­ing deal for hear­ing loss drug

A growing gene therapy company is teaming up with one of the largest gene therapy manufacturers to get its drug into patients in clinical trials.

Myrtelle announced on Monday that it inked a deal with Forge Biologics to make Myr-201, a gene therapy indicated for monogenic hearing loss. The target patient population, Myrtelle said, is people with a type of hearing loss called DFNB8, or autosomal recessive deafness 8.

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Take­da to pull key hy­poparathy­roidism drug from the mar­ket af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

Kaile Zagger, Infinant Health CEO

UC Davis mi­cro­bio­me spin­out re­brands in­fant sup­ple­ment busi­ness with na­ture fo­cus

When Kaile Zagger took the helm of UC Davis spinout Evolve Biosystems several months ago, the company billed itself as a probiotic maker.

However, she believes the company’s Evivo supplement designed to help infants develop a healthy gut microbiome is “so much more” — and that, she said, calls for a rebrand.

Evolve has, well, evolved into Infinant Health, the company announced on Monday. The new name is a mash-up of the words “infant” and “infinite,” representing the company’s goal of expanding beyond infant care. While its sole product, Evivo, is intended for newborns, Infinant is “quickly developing” an option for kids through the age of two.

Leo Tarkovsky, Fingerpaint Group's new chief commercial officer

Fin­ger­paint Group taps for­mer WPP and Mc­Cann Health ex­ec for new com­mer­cial role

Healthcare agency veteran Leo Tarkovsky has joined Fingerpaint Group as chief commercial officer to oversee its growing portfolio of pharma and healthcare agencies and communications companies.

Tarkovsky came to Fingerpaint from WPP where he was EVP for global healthcare growth over the past year. Before that served at McCann Health for seven years including as president overseeing the New York agencies with pharma clients including AstraZeneca, Bristol Myers Squibb, Eli Lilly and Johnson & Johnson’s Janssen.

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