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

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.