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.

Ifen­prodil, al­so known as NP-120, is an N-methyl-D-as­par­tate (NM­DA) re­cep­tor an­tag­o­nist. NM­DA re­cep­tors are found on many tis­sues, in­clud­ing lung cells, and are tar­gets among painkillers and anes­thet­ics. How­ev­er, Al­ger­non touts ifen­prodil as a po­ten­tial first-in-class op­tion for IPF and chron­ic cough.

Ac­cord­ing to up­dat­ed Phase IIa da­ta re­leased back in Ju­ly, ifen­prodil helped re­duce pa­tients’ mean 24-hour cough counts by 32% at 4 weeks (p = 0.023) and 39.5% at 12 weeks (p = 0.001) com­pared to base­line.

Christo­pher More­au

“We ap­pre­ci­ate the U.S. FDA’s de­ci­sion to grant ODD sta­tus to Ifen­prodil for IPF, a dis­ease for which prog­no­sis re­mains dis­mal, with 50% mor­tal­i­ty ex­pect­ed with­in 3-4 years,” Al­ger­non CEO Christo­pher More­au said in a news re­lease.

The news comes as the FDA is “not cur­rent­ly fi­nal­iz­ing ODE [or­phan drug ex­clu­siv­i­ty] de­ter­mi­na­tions” be­cause of the “far-reach­ing im­pli­ca­tions” of a re­cent court case in­volv­ing Cat­a­lyst and Ja­cobus. A US ap­peals court over­turned a pri­or FDA court win, rul­ing that the agency shouldn’t have ap­proved a rare dis­ease drug be­cause a pre­vi­ous­ly ap­proved (and more ex­pen­sive) one with the same in­gre­di­ent had or­phan drug ex­clu­siv­i­ty.

The FDA told End­points News in a state­ment last week:

The de­ci­sion has caused un­cer­tain­ty for rare dis­ease drug de­vel­op­ment. Un­der the court’s in­ter­pre­ta­tion of the scope of ODE, ODE would block ap­proval of an­oth­er com­pa­ny’s ap­pli­ca­tion for the same drug for the en­tire dis­ease or con­di­tion for which the drug is grant­ed or­phan-drug des­ig­na­tion, re­gard­less of whether the drug was ap­proved on­ly for a nar­row­er use or in­di­ca­tion. Un­der that in­ter­pre­ta­tion, a spon­sor could seek ap­proval and ex­clu­siv­i­ty for a drug by fo­cus­ing on the small­est, eas­i­est-to-study pop­u­la­tions and such ex­clu­siv­i­ty would block the drug for the en­tire dis­ease, even though the spon­sor did not in­vest in study­ing and de­vel­op­ing the drug for all in­di­vid­u­als with the dis­ease.

The agency told End­points News on Mon­day that it had no up­dates to pro­vide.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

Post-hoc analy­sis: EMA's CHMP re­jects Ipsen's po­ten­tial drug for rare ge­net­ic dis­ease

The European Medicines Agency’s Committee for Medicinal Products for Human Use on Friday rejected Ipsen Pharma’s potential treatment for a rare genetic disease known as fibrodysplasia ossificans progressiva (FOP), which causes extra bone to form outside the skeleton.

The EMA said on its website that it could not draw any firm conclusions on the benefits of the French biopharma’s Sohonos (palovarotene), which selectively targets the retinoic-acid receptor gamma (RARγ), “as the applicant’s conclusion was based on a post-hoc analysis which was neither scientifically nor clinically justified and pre-specified study objectives were not met.”

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FDA ap­proves an­oth­er in­di­ca­tion for Keytru­da, this time in the ad­ju­vant NSCLC set­ting

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.