E&C chair Frank Pallone (Kevin Dietsch/Pool via AP Images)

Ac­cel­er­at­ed ap­proval re­forms take cen­ter stage at House hear­ing, but a way for­ward re­mains un­clear

The House En­er­gy & Com­merce com­mit­tee’s health sub­com­mit­tee on Thurs­day dug in­to al­most two dozen dif­fer­ent FDA- and phar­ma-re­lat­ed bills that may be tacked on­to a larg­er bill or even the bio­phar­ma user fee bills, which have to be reau­tho­rized be­fore the end of Sep­tem­ber.

The mas­sive batch of bills mak­ing their way through the House in­cludes two crit­i­cal bills that seek to re­form the FDA’s ac­cel­er­at­ed ap­proval process in dif­fer­ent ways.

On the one hand is E&C chair Frank Pal­lone (D-NJ), who, to the cha­grin of in­dus­try, is seek­ing ex­pi­ra­tion dates (5 years max) on ac­cel­er­at­ed ap­provals giv­en the fact that com­pa­nies of­ten drag their feet on con­fir­ma­to­ry tri­als. His bill is run­ning in­to op­po­si­tion from Re­pub­li­can Cathy Mc­Mor­ris Rodgers of Wash­ing­ton, who sought to make clear the cur­rent ac­cel­er­at­ed path­way is func­tion­ing well, but that the path­way needs to be mod­ern­ized for some new drugs, like those for ALS, that don’t have a clear sur­ro­gate end­point.

Mc­Mor­ris Rodgers’ bill would make it so that the ev­i­dence used to sup­port that an end­point is rea­son­ably like­ly to pre­dict a clin­i­cal ben­e­fit un­der an ac­cel­er­at­ed ap­proval “may in­clude epi­demi­o­log­i­cal, patho­phys­i­o­log­i­cal, ther­a­peu­tic, phar­ma­co­log­ic, or oth­er ev­i­dence de­vel­oped us­ing bio­mark­ers, for ex­am­ple, or oth­er sci­en­tif­ic meth­ods or tools.”

Pal­lone, mean­while, of­fered the ex­am­ple of Bio­gen’s con­tro­ver­sial new Alzheimer’s drug Aduhelm, which won an ac­cel­er­at­ed ap­proval last June.

“Here we are nine months lat­er, and the spon­sor has not screened a sin­gle pa­tient for its re­quired con­fir­ma­to­ry tri­al,” Pal­lone said. “Oth­er drugs have stayed on the mar­ket for eight or nine years with­out prov­ing a clin­i­cal ben­e­fit.”

He al­so not­ed how CDER Di­rec­tor Pa­trizia Cavaz­zoni tes­ti­fied last month on how “the process for re­mov­ing these drugs from the mar­ket is cum­ber­some and can take months or even years.”

Rank­ing mem­ber Rep. Brett Guthrie (R-KY), how­ev­er, took the oth­er side on the Aduhelm de­bate, say­ing that CMS’ de­ci­sion to re­strict cov­er­age to on­ly clin­i­cal tri­als could have a chill­ing ef­fect on oth­er in­vest­ments in Alzheimer’s.

Sup­port­ing the ac­cel­er­at­ed ap­proval sta­tus quo, Jeff Allen, CEO of Friends with Can­cer Re­search, not­ed that ac­cel­er­at­ed ap­provals con­vert­ing to full ap­proval took a me­di­an time of 3.1 years. He al­so said that if ac­cel­er­at­ed ap­proval drugs are in­ap­pro­pri­ate­ly lim­it­ed, there could be de­lays in ac­cess, but the hall­mark of the ac­cel­er­at­ed path­way is bal­anc­ing these un­cer­tain­ties.

“I think the leg­is­la­tion pro­posed to­day to strength­en the post-mar­ket sur­veil­lance side will help to ex­pand the de­vel­op­ment of sur­ro­gate end­points,” he not­ed.

Resh­ma Ra­machan­dran, physi­cian-fel­low in Yale’s Na­tion­al Clin­i­cian Schol­ars Pro­gram, tes­ti­fied that phar­ma com­pa­nies of­ten mar­ket drugs via the ac­cel­er­at­ed path­way for long pe­ri­ods of time pri­or to the con­fir­ma­to­ry tri­al re­sults. And some­times that mar­ket­ing con­tin­ues even af­ter con­fir­ma­to­ry tri­als have failed.

She not­ed that Pal­lone’s bill builds in flex­i­bil­i­ty for FDA on when ap­pro­pri­ate con­fir­ma­to­ry tri­al com­ple­tion date can be set, and it al­lows for ne­go­ti­a­tions be­tween the spon­sor and FDA. That would mean drugs won’t be au­to­mat­i­cal­ly with­drawn if such a drug is still in a con­fir­ma­to­ry tri­al.

In ad­di­tion to ac­cel­er­at­ed ap­provals, the bills dis­cussed at the hear­ing al­so in­clude fur­ther fund­ing and struc­ture for ARPA-H, a new NIH-housed agency (with $1 bil­lion in starter funds) that will cor­rect the gap be­tween the ba­sic re­search at NIH and the de­vel­op­ment of prod­ucts in the pri­vate sec­tor, ac­cord­ing to health sub­com­mit­tee chair An­na Es­hoo (D-CA). Es­hoo al­so praised the years’ worth of work in the lat­est it­er­a­tion of the 21st Cen­tu­ry Cures Act.

Rep. Fred Up­ton (R-MI), who co-spon­sored both Cures bills with Rep. Di­ana DeGette (D-CO), not­ed on Thurs­day that Covid “has taught us a valu­able les­son that FDA can work quick­ly and that RWE will im­prove its de­ci­sion­mak­ing.”

Three oth­er bills dis­cussed would seek to im­prove clin­i­cal tri­al di­ver­si­ty, while oth­ers will help with gener­ic and biosim­i­lar de­vel­op­ment, in­clud­ing one that will al­low some gener­ics to be ap­proved with a “tem­porar­i­ly dif­fer­ent” la­bel than its ref­er­ence prod­uct.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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