Scott Got­tlieb is clos­ing a big loop­hole in the or­phan drug law

FDA com­mis­sion­er Scott Got­tlieb an­nounced Tues­day that the agency will re­lease guid­ance to close a loop­hole that al­lows com­pa­nies to avoid their oblig­a­tion to study phar­ma­ceu­ti­cals in pe­di­atric pop­u­la­tions.

The sit­u­a­tion, ac­cord­ing to Got­tlieb, aris­es if spon­sors re­ceived an or­phan des­ig­na­tion for a pe­di­atric sub­type of an oth­er­wise com­mon and non-or­phaned adult dis­ease.

For ex­am­ple, with a con­di­tion like in­flam­ma­to­ry bow­el dis­ease, a drug may be ap­proved to treat the large pop­u­la­tion of adults with the con­di­tion but then the same drug may be grant­ed an or­phan des­ig­na­tion to treat a sub­set of chil­dren suf­fer­ing from IBD.

“But once a drug re­ceives an or­phan des­ig­na­tion for a pe­di­atric pop­u­la­tion of the adult dis­ease, the drug then be­comes statu­to­ri­ly ex­empt from the re­quire­ments” of the Pe­di­atric Re­search Eq­ui­ty Act (PREA), Got­tlieb ex­plains.

“It’s a loop­hole that is in di­rect op­po­si­tion to what Con­gress in­tend­ed,” he wrote. “No­body en­vi­sioned this un­in­tend­ed con­flict be­tween the orig­i­nal ODA [Or­phan Drug Act] and the pro­vi­sions out­lined in PREA. In ef­fect, by let­ting spon­sors des­ig­nate pe­di­atric sub­pop­u­la­tions of drugs in­tend­ed to treat adult dis­eases, the drug mak­ers re­ceive an un­in­tend­ed ‘free pass’ from hav­ing to study drugs in these or oth­er pe­di­atric us­es. Thus, rather than en­sur­ing more pe­di­atric re­search, as Con­gress en­vi­sioned, we can end up with few­er pe­di­atric stud­ies. FDA will soon is­sue a draft guid­ance doc­u­ment that’s aimed at clos­ing this in­ad­ver­tent loop­hole.”

Nan­cy Good­man of Kids v Can­cer, whose RACE for Chil­dren’s Act was in­clud­ed in the FDA user fee reau­tho­riza­tion law, told me that the lan­guage on clos­ing the loop­hole was ini­tial­ly go­ing to be in­clud­ed in FDARA. It’s un­clear why it didn’t make it in the fi­nal lan­guage.

A pub­lic meet­ing an­nounced by Got­tlieb will al­so ad­dress some of the statutes from FDARA and get in­put on com­plex sci­en­tif­ic and reg­u­la­to­ry is­sues, such as those raised by mol­e­c­u­lar­ly tar­get­ed drugs and bi­o­log­ics and the ap­pro­pri­ate ap­pli­ca­tion of or­phan in­cen­tives.

In ad­di­tion to clos­ing that loop­hole and the meet­ing, Got­tlieb said FDA has now re­viewed all or­phan drug des­ig­na­tion re­quests old­er than 120 days and will put in place new poli­cies to im­prove the ef­fi­cien­cy of FDA’s re­view process to en­sure that we meet our new 90-day man­date to pre­vent new back­logs.


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.

Pho­to: AP Im­ages / end­points news

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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