Gaurav Shah, Rocket Pharmaceuticals CEO

FDA lifts clin­i­cal hold on Rock­et's gene ther­a­py for heart fail­ure af­ter 3-month de­lay

New Jer­sey’s Rock­et Phar­ma­ceu­ti­cals hit a ma­jor road­block for its AAV-de­liv­ered gene ther­a­py for heart fail­ure in May af­ter the FDA came ask­ing for more de­tails on tar­get pa­tients. Three months down the road, Rock­et is clear for liftoff once again.

The FDA on Mon­day lift­ed its clin­i­cal hold on Rock­et’s Phase I tri­al for gene ther­a­py can­di­date RP-A501 for Danon dis­ease, an ul­tra-rare ge­net­ic dis­or­der that can lead to heart fail­ure in chil­dren and young adults, the com­pa­ny said in a re­lease.

It’s been a lit­tle more than three-month wait for Rock­et af­ter the agency ground­ed the tri­al, ask­ing for a re­vised pro­to­col on “pa­tient se­lec­tion and man­age­ment.”

On Mon­day, Rock­et said it had ad­dressed those is­sues and was ready to move for­ward, cit­ing no new safe­ty red flags churned up dur­ing the agency’s re­view. The com­pa­ny ex­pects to re­sume dos­ing in the adult co­hort “as soon as pos­si­ble” and is gear­ing up to be­gin en­roll­ment for a pe­di­atric co­hort as well, Rock­et said.

Un­like the rest of its pipeline, which us­es lentivi­ral vec­tors to trans­port its gene ther­a­pies, RP-A501 is Rock­et’s on­ly can­di­date that us­es AAV9 for de­liv­ery, a vec­tor that has re­ceived in­tense scruti­ny af­ter a wave of safe­ty flags in re­cent years. The ther­a­py us­es that vec­tor to trans­port a func­tion­al ver­sion of lyso­some-as­so­ci­at­ed mem­brane pro­tein 2 (LAMP-2) in­to pa­tients’ cells — the mu­tat­ed form of the pro­tein caus­es a buildup of au­tophago­somes and glyco­gen in the heart and oth­er tis­sues that can cause heart fail­ure and death, par­tic­u­lar­ly in boys and young men.

Rock­et be­lieves there are some­where be­tween 15,000 and 30,000 pa­tients with Danon in the US and Eu­rope. The drug would be­come the first gene ther­a­py ap­proved for mono­genic heart fail­ure, Rock­et said.

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.”

FDA calls for non­in­fe­ri­or­i­ty tri­als for an­tibac­te­ri­als, thanks to new drugs for re­sis­tant in­fec­tions

The FDA on Monday said it updated its recommendations for the clinical development of new antibacterial drugs, and thanks to a new clutch of drugs, sponsors will have to conduct noninferiority trials that include subjects with infections caused by certain drug-resistant organisms because an effective active control can be provided.

The guidance, which is likely to be released within the next day or so, builds on a 2017 document and specifies what noninferiority trial designs may be used “with a wider NI margin,” including cases in which the trial population is enriched for subjects with infections caused by certain drug-resistant organisms, FDA said.

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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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).

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.

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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