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.

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”

Feud be­tween two biotechs left near­ly 12M dos­es of mon­key­pox an­tivi­ral on the ta­ble

As the US embarks on a new delivery strategy to stretch out its thinning supply of monkeypox vaccines, the need for treatments could pick up as cases of the virus rise. And the amount of courses of one potential antiviral, soon to be clinically tested for efficacy in humans, was almost 12 million more than it is today, according to SEC filings.

While not greenlit for treating monkeypox, SIGA’s FDA-approved smallpox antiviral, Tpoxx, can be requested by physicians under an expanded use program. As of Monday, HHS tells Endpoints News it had tapped into more than 15,000 of the 1.7 million courses of Tpoxx that have been stockpiled, but with cases climbing over the past few weeks, demand will likely not peter out in the near future, especially if the vaccine supply runs dry.

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HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.