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Christophe Weber, Takeda CEO (Miho Takahashi/The Yomiuri Shimbun via AP Images)

Af­ter a rocky decades-long jour­ney, Take­da’s CMV drug gets FDA OK

After a wild two-decade ride, the FDA has approved Takeda’s antiviral maribavir — now marketed as Livtencity — for cytomegalovirus, one of the most common viral infections experienced by transplant patients.

Takeda picked up maribavir a couple years ago in its $62 billion Shire buyout, at which point the drug had already failed a Phase III trial and changed hands a few times. But on Tuesday, Takeda touted a regulatory win in patients 12 years and older, marking the second approval this year for one of the company’s new molecular entities.

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Neil Desai, Aadi Bioscience CEO (via YouTube)

FDA ap­proves pricey new treat­ment for ul­tra-rare and ag­gres­sive form of sar­co­ma

The FDA on Tuesday approved Aadi Bioscience’s first drug and the first treatment approved specifically for patients with an ultra-rare and aggressive form of sarcoma that occurs mostly in women.

The approval of the drug, known as Fyarro, is for those with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa), and is based on a Phase II trial.

Results showed an overall response rate as assessed by independent review of 39% (12/31), with two patients achieving a complete response after prolonged follow up, Aadi said. The company also said that among responders, 92% had a response lasting greater than or equal to six months; 67% had a response lasting greater than or equal to 12 months; and 58% had a response lasting greater than or equal to two years.

Stephen Hahn and Donald Trump, AP Images

House com­mit­tee wants for­mer FDA com­mis­sion­er to come clean on Covid-re­lat­ed po­lit­i­cal in­ter­fer­ence

The House Select Subcommittee on the Coronavirus Crisis on Monday questioned former FDA commissioner Stephen Hahn on whether he acquiesced to political pressure in authorizing the use of hydroxychloroquine and convalescent plasma early in the pandemic, despite limited evidence of their effectiveness.

Hahn publicly disavowed any political interference in any of his agency’s EUA decisions, but behind the scenes, the pressure clearly caught up with him.

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Ugur Sahin (L) and Özlem Türeci (Bernd von Jutrczenka/picture-alliance/dpa/AP Images)

New Pfiz­er/BioN­Tech Covid vac­cine da­ta in teens show strong pro­tec­tion, lin­ing up sBLA fil­ing

Pfizer and BioNTech are planning to file for full approval of their Covid-19 vaccine for all of those over the age of 12 after they said longer-term analysis of the vaccine in teens continued to show strong protection against symptomatic cases of Covid-19 more than four months after the second dose.

With no serious safety concerns at least 6 months after the second dose for those ages 12 through 15, the companies said the data will form the basis for a planned upgrade from EUA to supplemental Biologics License Application (sBLA).

Jean-Jacques Bienaimé (BioMarin via Youtube)

FDA opens up an eth­i­cal can of worms with ac­cel­er­at­ed ap­proval for first drug for un­der­ly­ing ge­net­ic cause of dwarfism

The FDA on Friday signed off on an accelerated approval for BioMarin’s Voxzogo (vosoritide) injection, the first treatment to target the underlying genetics of dwarfism, which can increase the height of children five years of age and older with the condition.

The injection, which Biomarin said will have an annual net price of $240,000, works by binding to a specific receptor called the natriuretic peptide receptor-B, which reduces the growth regulation gene’s activity and stimulates bone growth in children.

FDA de­lays its de­ci­sion on Bris­tol My­er­s' $13B heart drug an­oth­er three months

Last week, Bristol Myers Squibb marched out long-term data for its heart drug mavacamten ahead of what execs had hoped would be a positive FDA decision in January. But regulators are saying they need a bit more time to think.

The FDA has extended mavacamten’s PDUFA date three months, from Jan. 28 to April 28, Bristol Myers announced on Friday. The news came just a few days after independent drug pricing watchdog ICER raised concerns about the candidate’s long-term safety in its final evidence report.

Michel Vounatsos (Biogen via YouTube)

Ex­clu­sive: FDA prob­ing death of Aduhelm pa­tient as Bio­gen's Alzheimer's drug con­tin­ues to stir con­tro­ver­sy

The FDA tells Endpoints News today that it’s aware of the death of a patient taking Biogen’s controversial Alzheimer’s drug Aduhelm (aducanumab) and is “actively investigating” the case.

Their statement follows the latest comments from RBC analyst Brian Abrahams, who is back with an update on the death, and this time he says that there are solid reasons to believe that the event was likely drug related and may have been preventable.

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Merck CEO Robert Davis

Mer­ck­'s Keytru­da scores first win in ad­ju­vant kid­ney can­cer, giv­ing it a leg up on check­point in­hibitor ri­vals

Merck is making room for yet another use on its Keytruda label — this time, as the first adjuvant immunotherapy for certain renal cell carcinoma patients after they’ve had kidney surgery.

The FDA has approved Keytruda in the adjuvant setting three weeks before its goal date, marking the latest in a streak of label expansions and giving the PD-1 superstar a leg up on its checkpoint inhibitor rivals.

FDA+ roundup: ALS bill would pro­vide $100M an­nu­al­ly to ac­cess po­ten­tial drugs; FDA needs to bet­ter en­force Clin­i­cal­Tri­als.gov re­port­ing, re­searchers say

The House Energy & Commerce Committee began marking up a dozen bills on Wednesday morning including one that would require the FDA to craft a five-year action plan for fostering the development of drugs that improve or extend the lives of people living with rare neurodegenerative diseases.

Rare neurodegenerative diseases, like amyotrophic lateral sclerosis or ALS, have been historically very difficult to treat. But this bipartisan bill, introduced by Rep. Mike Quigley (D-IL), will provide $100 million for each of fiscal years 2022 through 2026 to help HHS award grants to facilitate access to investigational drugs that diagnose or treat ALS.

Rep. Diana DeGette (D-CO) (Alex Brandon/AP Images)

Cures 2.0: Bi­par­ti­san House bill would al­low RWE to sat­is­fy con­fir­ma­to­ry tri­al re­quire­ments for ac­cel­er­at­ed ap­provals

Reps. Fred Upton (R-MI) and Diana DeGette (D-CO) on Tuesday unveiled the second iteration of their mammoth 21st Century Cures Act, a wide-ranging, mostly pro-pharma law signed by former President Obama that pushed the FDA in certain directions on real-world data and other hot button issues.

This time around there’s a provision to fund a $6.5 billion (on par with Biden’s request) research center at NIH to speed new treatments and make riskier investments. Similar to DARPA, the new NIH division to be known as ARPA-H, would be run by a small group of program managers with more latitude to pursue high-risk, high-reward projects.

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