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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Bris­tol My­ers Squibb sues No­var­tis for roy­al­ties sur­round­ing the use of trans­genic mice

Two Big Pharma companies are going to the mat over genetically modified mice in a licensing dispute.

Bristol Myers Squibb is suing Novartis in New York over a dispute concerning an evaluation, research and commercialization agreement stretching back to the late ’90s initially inked between Novartis and BMS’ predecessor Medarex. The deal in question allowed Novartis to use Medarex’s patented transgenic mice to develop therapeutic drugs. Novartis agreed to pay Medarex – and subsequently BMS – a royalty on sales of drugs it developed using the mice.

Anthony Fauci (AP Images)

West Vir­ginia man faces prison time for threat­en­ing emails to Fau­ci, oth­er health of­fi­cials

NIAID director Anthony Fauci gained hero status amid the pandemic, earning Americans’ trust and even Time magazine’s Guardian of the Year title. But he and other federal health officials have also faced intense threats, according to charges brought by the US Department of Justice.

A West Virginia man is facing up to 10 years in prison after threatening Fauci, former NIH director Francis Collins, and HHS assistant secretary for health Rachel Levine via email, the DOJ said on Monday. Thomas Patrick Connally, Jr., pleaded guilty on Monday to using an anonymous email address to threaten the officials for performing their official duties, including discussing Covid-19 testing and prevention.

Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.