Sarep­ta’s eteplirsen spurned as Eu­ro­pean reg­u­la­tors turn thumbs down on con­tro­ver­sial Duchenne MD drug

Sarep­ta’s ap­proval for eteplirsen — or Ex­ondys51 — in the US re­mains one of the most con­tro­ver­sial OKs in FDA his­to­ry, earn­ing a rare la­bel that says the biotech has yet to pro­duce ev­i­dence the drug works.

But light­ning won’t strike twice on the reg­u­la­to­ry path­way for this biotech.

The biotech re­port­ed Thurs­day af­ter­noon that the CHMP is prep­ping a neg­a­tive vote, bar­ring the drug from the con­ti­nent. And Sarep­ta shares im­me­di­ate­ly tum­bled 5% in af­ter-mar­ket trad­ing.

Doug In­gram

The drug, which has a sol­id set of sup­port­ers in the DMD com­mu­ni­ty, is get­ting snubbed de­spite the Eu­ro­peans’ ac­cep­tance of PTC Ther­a­peu­tics’ ri­val drug, which has failed re­peat­ed piv­otal stud­ies. But in DMD, reg­u­la­tors have a ten­den­cy to make things up as they go along, fre­quent­ly bend­ing the rules to ac­com­mo­date a pas­sion­ate group of pa­tients and par­ents — or not. PTC has been re­peat­ed­ly slapped down by the FDA, even af­ter Janet Wood­cock over­ruled a host of agency in­sid­ers with her in­sis­tence on ap­prov­ing eteplirsen.

Sarep­ta CEO Doug In­gram had this to say:

Un­for­tu­nate­ly, the CHMP’s trend vote was neg­a­tive. Based on dis­cus­sions with CHMP rep­re­sen­ta­tives, it is our un­der­stand­ing that the CHMP did not con­clude that eteplirsen is in­ef­fec­tive for ex­on 51 amenable pa­tients, but rather that Sarep­ta has not yet met the reg­u­la­to­ry thresh­old for con­di­tion­al ap­proval, in part due to the use of ex­ter­nal con­trols as com­para­tors in the stud­ies. Sarep­ta plans to file for re-ex­am­i­na­tion and will re­quest that a Sci­en­tif­ic Ad­vi­so­ry Group (SAG), which is made up of DMD and neu­ro­mus­cu­lar spe­cial­ists, be con­vened to pro­vide ex­pert guid­ance and in­sight in­to, among oth­er things, the va­lid­i­ty of the ex­ter­nal con­trols used and the im­por­tance of slow­ing pul­monary de­cline in pa­tients with DMD.

Hop­ing to take some of the sting out of the news, while dis­ap­point­ing some an­a­lysts with its Q1 per­for­mance, Sarep­ta al­so an­nounced a deal to part­ner with My­onexus Ther­a­peu­tics on its work de­vel­op­ing gene ther­a­pies for Limb-gir­dle mus­cu­lar dy­s­tro­phies. Sarep­ta paid $60 mil­lion up­front and of­fered $45 mil­lion more in mile­stones for the deal, which al­so pro­vides the biotech with a buy­out op­tion at proof-of-con­cept.

“My­onexus and its fo­cus on gene ther­a­py us­ing the AAVrh.74 vec­tor to treat forms of LGMD aligns bril­liant­ly with our vi­sion to emerge as one of the most mean­ing­ful glob­al pre­ci­sion ge­net­ic med­i­cine com­pa­nies by fo­cus­ing on the use of ge­net­ic med­i­cine to im­prove the lives of those with rare fa­tal dis­eases,” said In­gram in a pre­pared state­ment.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

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.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.

Phillip Gomez, SIGA CEO

UP­DAT­ED: On the back of SIGA Tech­nolo­gies' win with the FDA, the mon­key­pox virus sees the com­pa­ny spring­ing to fur­ther ac­tion

As the cases of monkeypox now sit at well over 100 worldwide and have spread to multiple continents, the orders for any type of vaccine against monkeypox are seeing nations and medical bodies looking to get their hands on anything and everything. And now SIGA Technologies seems to be getting in on the action.

According to Euronews, SIGA Technologies, a pharmaceutical company that is focused on providing medical countermeasures to biological and chemical attacks, is now in talks with several European authorities looking to stockpile its antiviral that can counter monkeypox. The drug known as tecovirimat or Tpoxx was approved by the FDA in 2018 as a vaccine for smallpox but was approved by the European Medicines Agency to also act against monkeypox, cowpox and complications from immunization with vaccinia.

Lutz Hegemann, Novartis president of global health

No­var­tis li­cens­es out leukemia drug as part of new glob­al coali­tion to in­crease ac­cess to can­cer treat­ments

The Union for International Cancer Control (UICC) has gathered a slate of Big Pharmas for its new collaboration in hopes of increasing access to cancer medicines in lower income countries, UICC announced yesterday.

Dubbed ATOM, or Access to Oncology Medicines, the coalition includes AstraZeneca, BeiGene, Novartis, Bristol Myers Squibb, Roche, Gilead, and Sanofi, among other organizations. The goal of the partnership is to increase generic and biosimilar development of cancer drugs as well as license out essential medicines to these countries. The third part of the partnership includes building up the infrastructure to diagnose cancers and properly handle cancer medicines.

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Bris­tol My­ers dusts off an old Cel­gene chemother­a­py for use in chil­dren with a rare blood can­cer

Bristol Myers Squibb is bringing new life to a chemotherapy drug from the old Celgene pipeline as it touts another approval in a rare form of blood cancer affecting young children.

The FDA on Friday approved Vidaza (chemically known as azacitidine) for pediatric patients 1 month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML). The new indication marks the first approval for Vidaza in more than a decade, though it’s commonly used to treat acute myeloid leukemia and myelodysplastic syndromes (MDS).