Night­star's drug for rare vi­sion loss ad­mit­ted to FDA's gene ther­a­py speed­way

In the lat­est show of reg­u­la­to­ry sup­port for gene ther­a­pies, Ox­ford spin­out Night­star Ther­a­peu­tics just got ac­cess to a fair­ly new “fast-lane” to speed its reti­nal gene ther­a­py to mar­ket.

The Lon­don-based com­pa­ny scored an FDA des­ig­na­tion called Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­py, or RMAT, for its Phase III drug NSR-REP1. The ther­a­py is de­signed to treat choroi­deremia, a rare ge­net­ic con­di­tion that caus­es pro­gres­sive vi­sion loss, by cor­rect­ing mu­ta­tions in the CHM gene.

David Fel­lows

“Re­ceiv­ing RMAT des­ig­na­tion for NSR-REP1 high­lights the po­ten­tial of this gene ther­a­py to main­tain and im­prove vi­su­al acu­ity in choroi­deremia,” said Dave Fel­lows, Night­star’s CEO, in a state­ment. “This des­ig­na­tion fur­ther un­der­scores a recog­ni­tion of the se­ri­ous na­ture of choroi­deremia and the ur­gent need to de­vel­op new treat­ments for those af­fect­ed by in­her­it­ed reti­nal dis­eases that would oth­er­wise lead to blind­ness. We look for­ward to work­ing close­ly with the FDA to dis­cuss the NSR-REP1 de­vel­op­ment pro­gram and to de­ter­mine how we can ac­cel­er­ate the path­way for mak­ing NSR-REP1 avail­able to choroi­deremia pa­tients.”

RMAT in­cludes all the ben­e­fits of the fast track and break­through des­ig­na­tions, in­clud­ing ear­ly in­ter­ac­tions be­tween the FDA and spon­sors. Back when RMAT was first cre­at­ed (as part of the 21st Cen­tu­ry Cures Act), drug­mak­ers wor­ried gene ther­a­pies would be ex­clud­ed. That’s be­cause the FDA didn’t ex­plic­it­ly in­clude gene ther­a­pies in the first de­scrip­tion of the des­ig­na­tion. The agency reme­died that, how­ev­er, last No­vem­ber.

Un­like the break­through des­ig­na­tion, RMAT doesn’t re­quire ev­i­dence that the drug may of­fer a sub­stan­tial im­prove­ment over avail­able ther­a­pies. And like break­through des­ig­na­tions, get­ting an RMAT des­ig­na­tion doesn’t mean the prod­uct will be ap­proved and does not change the stan­dards for safe­ty and ef­fec­tive­ness need­ed for ap­proval.

RMAT des­ig­na­tion for NSR-REP1 was based on clin­i­cal da­ta sup­port­ing the main­te­nance and im­prove­ment of vi­su­al acu­ity from com­plet­ed Phase I/II tri­als in choroi­deremia pa­tients, the com­pa­ny said. The drug is now in Phase III tri­als.

Night­star’s stock $NITE, which first list­ed on Nas­daq last Sep­tem­ber, is up near­ly 6% on the news.

 

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).