Alex Azar (Photo by Pete Marovich/Getty Images)

HHS seeks to pull Trump-era 'tick­ing time­bom­b' rule that would've put 95% of FDA reg­u­la­tions on the chop­ping block

The HHS is propos­ing to with­draw a Trump-era fi­nal rule that would have sun­set thou­sands of FDA and oth­er pub­lic health agency rules based on how long they’ve been in place.

The de­ci­sion fol­lows the fil­ing of a law­suit last March, in which sev­er­al non­prof­its al­leged: “The out­go­ing ad­min­is­tra­tion plant­ed a tick­ing time­bomb set to go off in five years un­less the HHS, be­gin­ning right now, de­votes an enor­mous amount of re­sources to an un­prece­dent­ed and in­fea­si­ble task.”

That task for the FDA, ac­cord­ing to the HHS, would be to re­view over 7,000 sec­tions of the CFR that were pro­mul­gat­ed by the agency and which are more than 10 years old, or would be­come more than 10 years old dur­ing the first five years the rule would be in ef­fect, rep­re­sent­ing over 95% of the FDA’s cur­rent reg­u­la­tions. The HHS al­so pre­vi­ous­ly de­layed the start of the Trump rule so that crit­i­cal re­sources weren’t di­vert­ed away from the pan­dem­ic to the re­view of fed­er­al reg­u­la­tions.

The HHS said Fri­day that, over­all, pulling the rule will save it more than $900 mil­lion over the next decade, and added sig­nif­i­cant de­tails on the ways in which for­mer HHS Sec­re­tary Alex Azar’s pre­vi­ous fi­nal rule was flawed:

Our think­ing is in­formed by a reeval­u­a­tion of the fac­tu­al premis­es and con­clu­sions in the SUN­SET fi­nal rule that are cen­tral to the De­part­ment’s analy­sis of the rule’s im­pli­ca­tions and ef­fects. In par­tic­u­lar, based on a re­analy­sis of the reg­u­la­to­ry im­pact of the rule, we now be­lieve that the rule like­ly rest­ed on a flawed un­der­stand­ing of the re­sources re­quired for this un­der­tak­ing, which im­pli­cates the like­li­hood that HHS reg­u­la­tions would ex­pire, and which in turn will re­quire the De­part­ment to make re­source al­lo­ca­tion de­ci­sions which could im­pede the De­part­ment’s abil­i­ty to car­ry out oth­er key pri­or­i­ties. That di­ver­sion of re­sources will like­ly im­pede ef­forts to adopt new rules to ad­dress na­tion­al pri­or­i­ties and ad­vance eq­ui­ty for all, in­clud­ing his­tor­i­cal­ly un­der­served and mar­gin­al­ized com­mu­ni­ties. It is there­fore po­ten­tial­ly in­con­sis­tent with the cur­rent Ad­min­is­tra­tion’s poli­cies that aim to em­pow­er agen­cies to use ap­pro­pri­ate tools to achieve those ends.

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.

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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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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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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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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