Drug reg­u­la­tors look to har­mo­nize how they tack­le in­no­va­tion

The In­ter­na­tion­al Coali­tion of Med­i­cines Reg­u­la­to­ry Au­thor­i­ties (ICM­RA) re­cent­ly re­leased a re­port on how var­i­ous reg­u­la­tors world­wide are work­ing to­geth­er to bet­ter iden­ti­fy and ad­dress fu­ture reg­u­la­to­ry chal­lenges posed by new cat­e­gories of ther­a­peu­tics, like cell and gene ther­a­pies, and new tools for drug de­vel­op­ment, such as ar­ti­fi­cial in­tel­li­gence (AI).

The re­port, which is part of a wider ef­fort to re­duce du­plica­tive work and in­crease har­mo­niza­tion among drug reg­u­la­tors from the US, Eu­rope, Japan and else­where, delves in­to the top­ic of hori­zon scan­ning for new in­no­va­tions, which for most reg­u­la­tors is “still in its in­fan­cy.” The re­port al­so ad­dress­es nov­el reg­u­la­to­ry path­ways, such as ex­pe­dit­ed path­ways, and oth­ers re­lat­ed to ear­ly en­gage­ment with stake­hold­ers, such as Health Tech­nol­o­gy As­sess­ment agen­cies.

“The pace of in­no­va­tion in med­ical de­vices has sur­passed med­i­cines due adapt­able ap­proach­es to the reg­u­la­tion of in­no­v­a­tive prod­ucts. While the ex­ist­ing nov­el li­cenc­ing path­ways for med­i­cines have seen pos­i­tive re­sults in pro­vid­ing time­ly pa­tient ac­cess, cur­rent and up­com­ing in­no­va­tions will con­tin­ue to chal­lenge reg­u­la­tors,” the re­port notes.

Eu­ro­pean, Japan­ese and Sin­ga­pore drug reg­u­la­tors al­so fur­ther ex­plored the top­ics of ad­di­tive man­u­fac­tur­ing, genome edit­ing and AI.

“The prod­ucts and tech­nolo­gies ex­plored in the case stud­ies show that many reg­u­la­to­ry sci­ence tools are miss­ing at present (e.g. off-tar­get ef­fects de­tec­tion meth­ods) and must be de­vel­oped in or­der for reg­u­la­tors to be able to as­sess the prod­ucts across their life-cy­cle. In ad­di­tion, new tech­nolo­gies (such as ad­di­tive man­u­fac­tur­ing) re­quire adap­ta­tion of the ex­ist­ing reg­u­la­to­ry frame­works, as they fa­cil­i­tate the pro­duc­tion of more com­plex prod­ucts at the point of care rather than in ded­i­cat­ed man­u­fac­tur­ing sites,” the re­port adds.

Reg­u­la­tors will need to train staffers on the new prod­uct types, but will al­so have to ac­cess out­side ex­perts (e.g., soft­ware en­gi­neers), who will re­quire reg­u­la­to­ry train­ing, as well. The agen­cies pro­posed de­vel­op­ing a ca­pac­i­ty frame­work for ex­per­tise as a com­mon re­source, fea­tur­ing the map­ping of the re­quired skills and ex­per­tise for var­i­ous types of prod­ucts or tech­nolo­gies to as­sist reg­u­la­tors in iden­ti­fy­ing their ca­pac­i­ty needs.

ICM­RA, which is al­so look­ing to es­tab­lish an in­for­mal in­no­va­tion net­work among ICM­RA mem­bers lat­er this year, will seek to bet­ter ar­tic­u­late the com­mon char­ac­ter­is­tics of nov­el reg­u­la­to­ry path­ways and to as­sist the analy­ses and con­sid­er­a­tions of new and ex­ist­ing reg­u­la­to­ry meth­ods.

In ad­di­tion to the strate­gic pri­or­i­ty project re­port on in­no­va­tion, ICM­RA re­cent­ly re­leased a frame­work for the in­volve­ment of health au­thor­i­ties in the man­age­ment of glob­al health crises.

In­no­va­tion Strate­gic Pri­or­i­ty Pro­ject Re­port


First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Im­age: Shut­ter­stock

Fangliang Zhang, AP Images

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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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.

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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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.

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.

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.

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.