House pass­es 'right-to-try' — al­low­ing the ter­mi­nal­ly ill to ask com­pa­nies for ex­per­i­men­tal drugs out­side of FDA's purview

The House on Tues­day af­ter­noon passed a Sen­ate-rat­i­fied ver­sion of a bill, known as “right-to-try,” and Pres­i­dent Don­ald Trump has in­di­cat­ed he will sign it — fur­ther proof that the Koch broth­ers-backed Gold­wa­ter In­sti­tute’s politi­ciza­tion of the US Food and Drug Ad­min­is­tra­tion (FDA) is just be­gin­ning.

Rid­ing on the backs of ter­mi­nal­ly ill pa­tients who have run out of op­tions, Re­pub­li­cans pegged the bill as a last-ditch ef­fort to “try any­thing.” At an ear­li­er hear­ing, Rep. Mor­gan Grif­fith (R-VA) even went so far as to say that he would in­ject mon­key urine in­to him­self if “faced with one of these heart-rend­ing sit­u­a­tions.”

The bill’s back­ers, though, have yet to pub­licly dis­close why the pro­gram is specif­i­cal­ly nec­es­sary, or if there are ex­am­ples of ter­mi­nal­ly ill pa­tients who will gain ac­cess to in­ves­ti­ga­tion­al treat­ments be­cause of “right-to-try” (com­pa­nies are still al­lowed to re­ject re­quests) and would not have un­der FDA’s ex­pand­ed ac­cess pro­gram.

The House’s pas­sage of the Sen­ate bill from last Au­gust — af­ter a pre­vi­ous House bill passed but was not tak­en up by the Sen­ate, and af­ter a pre­vi­ous House ver­sion failed to pass — al­so fol­lows the right-to-try move­ment’s spread across state leg­is­la­tures. Pres­i­dent Don­ald Trump has said he would sign the bill and Vice Pres­i­dent Mike Pence said it was a pri­or­i­ty for him.

But Rep. Frank Pal­lone (D-NJ) called the bill “dan­ger­ous,” “not nec­es­sary” and said it “need­less­ly ex­pos­es pa­tients to un­known risks.” This bill is worse than what the House passed pri­or, Pal­lone said, not­ing a loop­hole in it that could ex­pand the num­ber of peo­ple than can use in­ves­ti­ga­tion­al drugs and are not ter­mi­nal­ly ill.

Pub­lic health ex­perts, in­clud­ing FDA Com­mis­sion­er Scott Got­tlieb (though he has since said he backs the bill and that there’s lee­way for im­ple­men­ta­tion), had raised con­cerns on var­i­ous ver­sions of the bill, par­tic­u­lar­ly as FDA al­ready has its own ver­sion of “right-to-try,” known as ex­pand­ed ac­cess, and the agency ap­proves more than 99% of all ex­pand­ed ac­cess re­quests.

Crit­ics of the ex­pand­ed ac­cess pro­gram have said it’s un­nec­es­sar­i­ly bur­den­some, though FDA has made clear that it can re­spond to an emer­gency re­quest with­in 24 hours.

Re­gard­less, with a right-to-try win un­der its belt, Gold­wa­ter now has its sights on eas­ing the off-la­bel pro­mo­tion of med­i­cines and fur­ther ef­forts to un­der­mine the FDA.


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

Im­age: Shut­ter­stock

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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Thank you, next: Take­da hands Ovid $196M cash to rein back in Phase III-ready seizure drug, re­viv­ing bat­tered stock

Soticlestat made it.

Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones.

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Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.

Presage teams with Mer­ck on its Phase 0 test­ing; Kem­Pharm AD­HD drug wins ap­proval in chil­dren aged 6 and up

Seattle-based Presage Biosciences, which approaches drug development through its microdosing platform, has some new partnerships and cash to come with them.

Presage closed a $13 million financing round Tuesday, aiming to expand its network of clinical trial sites and advance development of its microdosing injection devices. They also closed partnership deals with Merck and Maverick Therapeutics.

The financing included $7 million from new investors, including the LabCorp Venture Fund, Bristol Myers Squibb, and InHarv Partners. An additional $6 million convertible note from Takeda Ventures will convert to equity.