GAO To Launch In­ves­ti­ga­tion Of FDA’s Or­phan Drug Pro­gram

Act­ing on a re­quest from three in­flu­en­tial U.S. sen­a­tors, the gov­ern­ment’s ac­count­abil­i­ty arm con­firmed that it will in­ves­ti­gate po­ten­tial abus­es of the Or­phan Drug Act.

The Gov­ern­ment Ac­count­abil­i­ty Of­fice still must de­ter­mine the full scope of what it will look in­to and the method­ol­o­gy to be used. De­ter­min­ing the scope will take some months, said Chuck Young, GAO’s man­ag­ing di­rec­tor for pub­lic af­fairs.

Sen­a­tor Or­rin Hatch

Ear­li­er this month, Sens. Or­rin Hatch (R-Utah), Chuck Grass­ley (R-Iowa) and Tom Cot­ton (R-Ark.) sent a let­ter to the GAO and raised the pos­si­bil­i­ty that reg­u­la­to­ry or leg­isla­tive changes might be need­ed “to pre­serve the in­tent of this vi­tal law” that gives drug­mak­ers lu­cra­tive in­cen­tives to de­vel­op drugs for rare dis­eases.

Grass­ley’s of­fice said Tues­day they ex­pect­ed the GAO to be­gin its work in about nine months. The de­lay is typ­i­cal as the agency has a queue of re­quests it is pur­su­ing.

The sen­a­tors have asked the GAO to “in­ves­ti­gate whether the ODA is still in­cen­tiviz­ing prod­uct de­vel­op­ment for dis­eases with few­er than 200,000 af­fect­ed in­di­vid­u­als, as in­tend­ed.”

Con­gress over­whelm­ing­ly passed the 1983 Or­phan Drug Act to mo­ti­vate phar­ma­ceu­ti­cal com­pa­nies to de­vel­op drugs for peo­ple whose rare dis­eases had been ig­nored. Drugs ap­proved as or­phans are grant­ed tax in­cen­tives and sev­en years of ex­clu­sive rights to mar­ket drugs that are need­ed by few­er than 200,000 pa­tients in the U.S.

In re­cent months, re­ports of five- and six-fig­ure an­nu­al price tags for or­phan drugs have am­pli­fied long-sim­mer­ing con­cerns about abuse of the law. The sen­a­tors’ call for a GAO in­ves­ti­ga­tion re­flects that sen­ti­ment.

“While few will ar­gue against the im­por­tance of the de­vel­op­ment of these drugs, sev­er­al re­cent press re­ports sug­gest that some phar­ma­ceu­ti­cal man­u­fac­tur­ers might be tak­ing ad­van­tage of the mul­ti­ple des­ig­na­tion al­lowance in the or­phan drug ap­proval process,” the let­ter states.

In Jan­u­ary, Kaiser Health News pub­lished an in­ves­ti­ga­tion that found the or­phan drug pro­gram is be­ing ma­nip­u­lat­ed by drug­mak­ers to max­i­mize prof­its and to pro­tect niche mar­kets for med­i­cines be­ing tak­en by mil­lions.

That in­ves­ti­ga­tion, which al­so was pub­lished and aired by NPR, found that many drugs that now have or­phan sta­tus aren’t en­tire­ly new. More than 70 were drugs first ap­proved by the Food and Drug Ad­min­is­tra­tion for mass-mar­ket use. Those in­clude cho­les­terol block­buster Crestor, Abil­i­fy for psy­chi­atric dis­or­ders and the rheuma­toid arthri­tis drug Hu­mi­ra, the world’s best-sell­ing drug.

Oth­ers are drugs that have re­ceived mul­ti­ple ex­clu­siv­i­ty pe­ri­ods for two or more rare con­di­tions.

The sen­a­tors asked the GAO for a list of drugs ap­proved or de­nied or­phan sta­tus by the FDA. It al­so asked if re­sources at the FDA, which over­sees the law, have “kept up with the num­ber of re­quests” from drug­mak­ers and whether there is con­sis­ten­cy in the de­part­ment’s re­views.

And they said it would be im­por­tant to in­clude pa­tient ex­pe­ri­ences in the GAO re­view. The GAO does not pro­vide up­dates on on­go­ing work but rather re­ports its find­ings once they com­plete an as­sign­ment.

The rare-dis­ease drugs have be­come in­creas­ing­ly pop­u­lar with phar­ma­ceu­ti­cal and biotech com­pa­nies and are ex­pect­ed to com­prise 21.4 per­cent of world­wide pre­scrip­tion sales by 2022, not in­clud­ing gener­ics, ac­cord­ing to con­sult­ing firm Eval­u­atePhar­ma’s 2017 or­phan drug re­port.

That’s in part be­cause of the ex­or­bi­tant prices that can be charged. Of the top 100 drugs in the U.S., the av­er­age cost per pa­tient per year for an or­phan drug was $140,443 in 2016, com­pared with $27,756 for a non-or­phan, Eval­u­atePhar­ma said.


Orig­i­nal­ly pub­lished by Kaiser Health News. Kaiser Health News, a non­prof­it health news­room whose sto­ries ap­pear in news out­lets na­tion­wide, is an ed­i­to­ri­al­ly in­de­pen­dent part of the Kaiser Fam­i­ly Foun­da­tion.

Fangliang Zhang, AP Images

Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

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.

Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.

RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

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.

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.

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