FDA looks to strength­en ex­pand­ed ac­cess pro­gram

The FDA on Thurs­day un­veiled plans to fur­ther strength­en its ex­pand­ed ac­cess pro­gram, as the con­tro­ver­sial Right to Try leg­is­la­tion has yet to help any pa­tients.

The ex­pand­ed ac­cess pro­gram al­lows pa­tients with se­ri­ous or im­me­di­ate­ly life-threat­en­ing dis­eases who are un­able to par­tic­i­pate in clin­i­cal tri­als to gain ac­cess to promis­ing in­ves­ti­ga­tion­al treat­ments.

Over the last five years, the FDA has au­tho­rized more than 9,000 ap­pli­ca­tions across drugs, bi­o­log­ics and de­vices through the agency’s ex­pand­ed ac­cess pro­gram, and the agency ex­pects about 800 more in­di­vid­ual re­quests next year. About 99% of all the re­quests the FDA has re­ceived, across all ap­pli­ca­tion types, have been grant­ed, though those re­quests are grant­ed af­ter the com­pa­ny of­fer­ing the treat­ment al­so signs off on the re­quest.

Fol­low­ing an in­de­pen­dent as­sess­ment of the ex­pand­ed ac­cess pro­gram last year, the agency says that it has now agreed to up­date the FDA’s Ex­pand­ed Ac­cess web­pages to im­prove us­abil­i­ty through the stream­lin­ing of con­tent and a more user-friend­ly or­ga­ni­za­tion. “This in­cludes a re­duc­tion in du­pli­ca­tion, as well as the ad­di­tion of new pages with com­mon­ly re­quest­ed in­for­ma­tion, such as forms and key­words,” the FDA said.

And as part of the re­cent­ly an­nounced pro­posed re­or­ga­ni­za­tion of the Of­fice of the Com­mis­sion­er, the FDA al­so in­tends to for­mal­ly es­tab­lish an agency-wide Pa­tient Af­fairs Staff and Health Care Provider Af­fairs Pro­gram, un­der the over­sight of the Of­fice of Clin­i­cal Pol­i­cy and Pro­grams.

“This will en­hance our en­gage­ment with these im­por­tant ex­ter­nal stake­hold­er groups. The Pa­tient Af­fairs Staff is al­ready in place and charged with serv­ing as the ‘home base’ and pri­ma­ry point of en­try for pa­tients and physi­cians start­ing the EA process and nav­i­gat­ing them through the steps,” the FDA said.

Mean­while, the FDA has es­tab­lished an agency-wide Ex­pand­ed Ac­cess Co­or­di­nat­ing Com­mit­tee, which fa­cil­i­tates cross-cen­ter com­mu­ni­ca­tion and pro­motes dis­cus­sion to ad­dress cross-cut­ting is­sues re­lat­ed to ex­pand­ed ac­cess to pro­mote con­sis­ten­cy and best prac­tices.

Right to Try

Mean­while, the FDA has re­leased a new web­site to help pa­tients un­der­stand Right to Try, and the agency has es­tab­lished a work group to con­sid­er what steps may be re­quired to im­ple­ment this leg­is­la­tion in a way that ad­vances Con­gress’ in­tent to pro­mote ac­cess and pro­tect pa­tients.

But such im­ple­men­ta­tion ef­forts from the FDA have al­ready proven con­tro­ver­sial, as Sen. Ron John­son (R-WI) made clear that the law “in­tends to di­min­ish the FDA’s pow­er over peo­ple’s lives.

Fo­cus reached out to two com­pa­nies ad­ver­tis­ing the use of Right to Try – Stem­Vacs and Ther­a­peu­tic So­lu­tions In­ter­na­tion­al – though nei­ther re­spond­ed.

A re­cent ar­ti­cle in Health Af­fairs on Right to Try not­ed, “In a way, pa­tients may have ben­e­fit­ed from RTT af­ter all: not be­cause it cre­at­ed a new path­way that cut the FDA out of the pic­ture, but be­cause it raised aware­ness that non-tri­al ac­cess was pos­si­ble, thus gal­va­niz­ing pa­tients and their doc­tors to re­quest it.”


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.

Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

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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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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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.

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.

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.

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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.

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.