FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usu­al­ly re­served for mak­ing de­ci­sions on drug ap­pli­ca­tions or en­forc­ing what Con­gress stip­u­lates, the FDA is now dip­ping its toe in­to the wild world of con­gres­sion­al pol­i­tics as it at­tempts to fix a ma­jor court de­ci­sion that could have a chill­ing ef­fect on rare dis­ease R&D.

The case in ques­tion from last Oc­to­ber saw a US ap­peals court over­turn a pri­or FDA court win, say­ing that the agency nev­er should’ve ap­proved a rare dis­ease drug be­cause a pre­vi­ous­ly ap­proved but more ex­pen­sive drug with the same ac­tive in­gre­di­ent has or­phan drug ex­clu­siv­i­ty bar­ring such an ap­proval.

While seem­ing­ly straight­for­ward, the FDA now says the de­ci­sion could have cat­a­stroph­ic con­se­quences be­cause of the way the de­ci­sion ad­dress­es the scope of or­phan drug ex­clu­siv­i­ty, po­ten­tial­ly al­low­ing com­pa­nies to tar­get a tiny in­di­ca­tion for a spe­cif­ic dis­ease and then au­to­mat­i­cal­ly re­ceive 7 years of ex­clu­siv­i­ty for the en­tire dis­ease.

For in­stance, if the FDA ap­proves a drug for the “treat­ment of adult pa­tients with a par­tic­u­lar gene mu­ta­tion with cys­tic fi­bro­sis,” the ap­proval of the ap­pli­ca­tion will block the FDA from ap­prov­ing – for sev­en years (or­phan drug ex­clu­siv­i­ty) – an­oth­er com­pa­ny’s ap­pli­ca­tion “for the same drug for any in­di­ca­tion [bold is FDA’s] with­in cys­tic fi­bro­sis, in­clud­ing for chil­dren and for pa­tients with­out the par­tic­u­lar mu­ta­tion.”

In a rare state­ment on the court’s de­ci­sion, which FDA Law Blog points out is the equiv­a­lent of the agency lob­by­ing Con­gress, the agency makes clear that with­out con­gres­sion­al ac­tion:

In the com­ing months, the FDA will need to con­sid­er how the de­ci­sion af­fects drugs with ac­tive terms of or­phan drug ex­clu­siv­i­ty as well as cur­rent­ly mar­ket­ed drugs, in­clud­ing gener­ics. Go­ing for­ward, the FDA ex­pects that some drugs that are in late-stage de­vel­op­ment, or that have al­ready been sub­mit­ted for mar­ket­ing ap­pli­ca­tion re­view, would be blocked from ap­proval un­der the Cat­a­lyst de­ci­sion’s in­ter­pre­ta­tion of the [Or­phan Drug] Act.

The agency al­so ex­plains how this new court rul­ing in­cen­tivizes spon­sors to seek ap­proval and ex­clu­siv­i­ty for their drugs by fo­cus­ing on the small­est, eas­i­est-to-study pop­u­la­tions.

“Un­der the in­ter­pre­ta­tion in the Cat­a­lyst de­ci­sion, such an ap­proval would re­sult in ex­clu­siv­i­ty for their drug for the en­tire dis­ease, even though the spon­sors did not in­vest in study­ing and de­vel­op­ing their drug for all in­di­vid­u­als with the dis­ease,” the agency ex­plains.

Re­spond­ing to a ques­tion from Sen. Tam­my Bald­win (D-WI) on the is­sue, CDER di­rec­tor Pa­trizia Cavaz­zoni al­so re­cent­ly said she wants to work with Con­gress to find a so­lu­tion as this de­ci­sion “will send a chill in­to the de­vel­op­ment of rare dis­eases and it will dis­pro­por­tion­ate­ly af­fect chil­dren with rare dis­eases. It’s es­sen­tial to con­tin­ue to gen­er­ate the study of drugs in chil­dren, so this de­ci­sion will re­al­ly go counter to that.”

Bald­win and Sen. Bill Cas­sidy (R-LA) in­tro­duced bi­par­ti­san leg­is­la­tion last week, known as the Re­tain­ing Ac­cess and Restor­ing Ex­clu­siv­i­ty (RARE) Act, which would fix this court-re­lat­ed is­sue by cod­i­fy­ing the FDA’s long­stand­ing in­ter­pre­ta­tion of the Or­phan Drug Act of 1983 to en­sure that the scope of the or­phan ex­clu­siv­i­ty is clar­i­fied to ap­ply on­ly to the same ap­proved use or in­di­ca­tion with­in such rare dis­ease or con­di­tion in­stead of the same dis­ease or con­di­tion.

The FDA Law Blog al­so notes that as the world waits to hear whether or not the Supreme Court will take up the Cat­a­lyst case, the user fee reau­tho­riza­tion bill mak­ing its way through Con­gress al­so “pro­vides the per­fect ve­hi­cle for the Agency to try and get this ac­com­plished.”

And in­deed, a sec­tion of the bill marked up in the House, ti­tled “Clar­i­fy­ing ap­pli­ca­tion of ex­clu­sive ap­proval, cer­ti­fi­ca­tion, or li­cen­sure for drugs des­ig­nat­ed for rare dis­eases or con­di­tions,” would amend the statute to re­place the phrase “same dis­ease or con­di­tion” (and sim­i­lar phras­es) with “same ap­proved in­di­ca­tion or use with­in such rare dis­ease or con­di­tion” (and sim­i­lar phras­es).

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

FDA side­lines Paul Hud­son's $3.7B MS drug af­ter es­tab­lish­ing link to liv­er dam­age

One of Sanofi CEO Paul Hudson’s top picks in the pipeline — picked up in a $3.7 billion buyout 2 years ago — has just been sidelined in the US by a safety issue.

The pharma giant put out word early Thursday that the FDA has put their Phase III studies of tolebrutinib in multiple sclerosis and myasthenia gravis on partial clinical hold, halting enrollment and suspending dosing for patients who have been on the drug for less than 60 days. Patients who have completed at least 60 days of treatment can continue therapy as researchers explore a “limited” — but unspecified in Sanofi’s statement — number of cases of liver injury.

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Phar­ma re­acts to post-Roe; Drug­mak­ers beef up cy­ber de­fense; Boehringer, Roche qui­et­ly axe drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As a reminder, we are off on Monday for the Fourth of July. I hope this recap will kick off your (long) weekend well and that the rest of it will be just what you need. See you next week for a shortened edition!

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Eric Hughes, incoming Teva EVP of global R&D and CMO

Te­va chief raids Ver­tex for his new glob­al head of re­search and de­vel­op­ment

Teva CEO Kåre Schultz has found his new R&D chief and CMO in Vertex’s ranks.

The global generics giant, which has some 3,500 staffers in the R&D group, has named Eric Hughes to the top research spot in the company. He’ll be replacing Hafrun Fridriksdottir, who held the role for close to five years, on Aug. 1.

Hughes hasn’t been at Vertex for long, though. He jumped from Novartis less than a year ago, after heading the immunology, hepatology & dermatology global development unit. Before that, he completed a five-year stint as head of early clinical research for the specialty discovery medicine department in the exploratory clinical & translational research group at Bristol Myers Squibb, according to his LinkedIn profile.

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#BIO22: Man­ag­ing a biotech in tur­bu­lent times. 'There's a per­fect shit­show out there'

On Tuesday, June 14, Endpoints News EIC John Carroll sat down with a group of biotech execs to discuss the bear market for industry stocks and how they were dealing with it. Here’s the conversation, which has been lightly edited for brevity.

Martin Meeson, sponsor opening:

Thank you, John. Hello everyone. My name’s Martin Meeson, I’m the CEO of Fujifilm Diosynth. For those of you who don’t know Fujifilm Diosynth, we operate in the development of clinical and commercial product scale up, we have facilities in Europe and the US, and around about 4,000 employees. We run on average about 150 programs, so when it comes to managing in turbulent times over the last two years, we’ve had quite a lot of experience of that. Not just keeping the clinical pipelines and the commercial pipelines open, but also our response to the pandemic and the molecules that we’ve had within there. One of the phrases that I coined probably about a year ago when we were talking at JP Morgan, was I talked about managing through turbulent times. Well, it’s become the fact that we are not managing and leading through these times, we are managing in them, which is why that’s really the purpose of and the topic that we’ve got today.

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Emer Cooke, ICRMA chair (AP Photo/Geert Vanden Wijngaert)

ICM­RA to launch sev­er­al reg­u­la­to­ry pi­lot pro­grams cen­tered around man­u­fac­tur­ing in­spec­tions

As regulatory agencies look to catch up on inspections amid the Covid-19 pandemic, ICMRA is unveiling several pilot programs to address industry applications and inspections.

ICMRA, which is made up of the world’s top drug regulators, is launching multiple pilot programs, including two regulatory pilots addressing facility inspections for chemistry and manufacturing controls (CMC) and post-approval change (PAC) submission assessments and related regulatory actions.

FDA slaps warn­ing let­ter on Min­neso­ta API fa­cil­i­ty af­ter em­ploy­ee de­stroys clean­ing log

A manufacturing facility belonging to the Netherlands-based API producer Fagron Group has entered the FDA’s crosshairs after an employee destroyed a cleaning log, among other violations.

One of its plants in Saint Paul, MN received a warning letter on June 14, following an inspection last November that uncovered cross-contamination concerns.

“In your response, you provided a follow-up cleaning validation report in which you only assessed the carryover of niacin swab samples but not progesterone, which was included in your initial cleaning validation,” FDA says in the letter. “The lack of progesterone (b)(4) [commercially confidential information] is concerning considering the failing residue results you provided to investigators would yield unacceptable levels of progesterone cross-contamination.”

Amgen's taking social media followers around the globe as it introduces the many different

From Tam­pa to Mu­nich, Am­gen’s ‘Places’ cam­paign in­tro­duces its lo­ca­tions around the world

Amgen is taking social media followers around the world with its latest corporate campaign. Called “Places of Amgen,” the twice monthly posts highlight the biopharma’s different offices and sites – and the people who work there.

Each post runs on LinkedIn, Facebook and Instagram with details about the work Amgen does in that location, when it was established, comments from people who work there and other interesting facts. The most recent one about Paris, France, for example, notes that Amgen France last year signed a French association charter committed to the inclusion of LBGT+ people in the workplace.

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On Friday, Lonza announced plans to construct a large-scale commercial drug product fill and finish facility in the town of Stein, Switzerland.

Lon­za to in­vest $500M+ on fill-fin­ish fa­cil­i­ty on its home turf

Lonza has been expanding its reach across the globe, bringing sites in China and the US online this year, but now they are looking closer to home for their next major investment.

The Swiss manufacturer on Friday announced plans to construct a large-scale commercial drug fill and finish facility in the town of Stein, Switzerland. The new facility will be delivered through an investment of approximately CHF 500 million, or $519 million, and is expected to be completed in 2026. The facility will also be constructed on the same campus as Lonza’s current clinical drug product facility.

Credit: Shutterstock

Bio­haven takes mi­graine cam­paign to pa­tients' Twit­ter feeds, months ahead of Pfiz­er takeover

Two weeks ago, Biohaven hit an all-time high in weekly Nurtec prescriptions. CEO Vlad Coric attributes at least some of that success to a new interactive Twitter campaign that encourages patients to free their feed of potential migraine triggers.

Earlier this month, Biohaven in partnership with Twitter launched the #RelieveYourFeed campaign that allows users to customize their app settings based on their migraine triggers.