Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA/Sipa via AP Images)

Un­like­ly duo leads bi­par­ti­san Sen­ate bill to ex­pand Right to Try in­to MD­MA and psilo­cy­bin

Sens. Cory Book­er (D-NJ) and Rand Paul (R-KY) like­ly don’t share much in com­mon when it comes to pol­i­cy views, but that didn’t stop the pair from in­tro­duc­ing a new bill this week that would al­ter the con­tro­ver­sial Right to Try Act to al­low ter­mi­nal­ly ill pa­tients to ac­cess Sched­ule I drugs af­ter a Phase I tri­al has been com­plet­ed.

Build­ing off its pre­de­ces­sor, the bill would skirt around the FDA’s ex­pand­ed ac­cess pro­gram and the DEA’s reg­u­la­tions around con­trolled sub­stances, and al­low pa­tients to po­ten­tial­ly ac­cess Sched­ule I drugs — like MD­MA and psilo­cy­bin — much more eas­i­ly, as the sen­a­tors say these drugs “have shown ex­cep­tion­al promise in treat­ing a va­ri­ety of men­tal health con­di­tions, in­clud­ing sui­ci­dal de­pres­sion, anx­i­ety, and PTSD.”

But as with Right to Try, the FDA is not the bar­ri­er that many as­sume it to be when it comes to ac­cess­ing in­ves­ti­ga­tion­al drugs. The agency signs off on more than 99% of all ex­pand­ed ac­cess re­quests it re­ceives. It al­so re­mains un­clear how many have at­tempt­ed and failed to gain ac­cess to Sched­ule I sub­stances out­side of clin­i­cal tri­als or FDA’s ex­pand­ed ac­cess pro­gram.

Al­i­son Bate­man-House

Al­i­son Bate­man-House, as­sis­tant pro­fes­sor at New York Uni­ver­si­ty and an ex­pert on ex­pand­ed ac­cess, ex­plained to End­points News how this bill builds on oth­er state ex­pan­sions of RTT leg­is­la­tion, even as any doc­tor with the ap­pro­pri­ate li­cens­ing can pre­scribe con­trolled sub­stances.

“This is not an at­tack on the FDA, but just on reg­u­la­tions in gen­er­al, as they don’t care who’s reg­u­lat­ing when it comes to bar­ri­ers to ac­cess,” Bate­man-House said.

But she said she has heard that there’s been a lot of in­ter­est from pa­tients around clin­i­cal tri­als test­ing MD­MA and psilo­cy­bin, al­though some of the larg­er play­ers in this space al­ready have ex­pand­ed ac­cess pro­grams set up.

The Wis­con­sin-based Usona In­sti­tute, for in­stance, pro­vides cGMP psilo­cy­bin for clin­i­cal stud­ies and has a process set up where­by el­i­gi­ble aca­d­e­m­ic re­searchers can ac­cess the sup­plies to ad­vance sci­en­tif­ic and med­ical un­der­stand­ing of psy­che­delics.

“Re­cent stud­ies sug­gest that MD­MA and psilo­cy­bin could rep­re­sent an enor­mous ad­vance­ment in men­tal health and psy­chophar­ma­col­o­gy,” Book­er said in a state­ment. “Un­for­tu­nate­ly, many el­i­gi­ble pa­tients who ur­gent­ly need care do not cur­rent­ly have ac­cess to these promis­ing ther­a­pies.”

Com­pan­ion leg­is­la­tion will be in­tro­duced in the House by Reps. Earl Blu­me­nauer (D-OR) and Nan­cy Mace (R-SC).

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Ab­b­Vie sur­veys emo­tion­al im­pact of chron­ic leukemia con­di­tion, finds 'roller coast­er' of emo­tions

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.

Sen­ate Dems cling to a sim­ple ma­jor­i­ty to pass some of the biggest drug pric­ing re­forms ever

The Pharmaceutical Research and Manufacturers of America — and their fleet of drug industry lobbyists on Capitol Hill — are known for never losing.

Whenever a big drug pricing bill comes up, an army of the industry group’s lobbyists descend onto the Hill and either smash it outright or dismantle it piece by piece.

But for perhaps the largest drug pricing reforms ever enacted, after more than a decade of Congress trying and failing to allow Medicare to negotiate prescription drug prices, those same lobbyists and their biopharma clients were dealt a stunning blow on Sunday afternoon.