FDA hits Am­i­cus with de­lay on 'break­through' Pompe drug

AT-GAA, Am­i­cus Ther­a­peu­tics’ lead Phase III drug for Pompe dis­ease, was at the cen­ter of at­ten­tion dur­ing the Q1 call with in­vestors Mon­day. Ex­ecs talked about their high hopes for an ap­proval by the Ju­ly 29 PDU­FA date, and an­a­lysts were ask­ing ques­tions about launch prepa­ra­tion.

The FDA, though, needs more time to make a de­ci­sion.

Just af­ter the mar­ket closed on Tues­day, Am­i­cus put out word that the FDA has pushed back its re­view pe­ri­od by 90 days. The agency was deal­ing with two sep­a­rate ap­pli­ca­tions — a BLA for cipaglu­cosi­dase al­fa and an NDA for miglu­s­tat — for the two com­po­nents of AT-GAA, with PDU­FA dates sched­uled two months apart.

The de­lay marks an­oth­er speed bump for Am­i­cus, which was forced to call off a spin­out of its gene ther­a­py unit via a SPAC merg­er in the wake of a set­back.

By com­bin­ing a re­com­bi­nant hu­man acid α-glu­cosi­dase with an en­zyme sta­bi­liz­er, Am­i­cus had hoped it could bring a next-gen ap­proach to pa­tients with Pompe dis­ease. Al­though the drug, which was be­stowed break­through ther­a­py des­ig­na­tion, failed to beat Lu­mizyme, Sanofi’s en­zyme re­place­ment ther­a­py, in a Phase III tri­al, the biotech pushed ahead cit­ing (sta­tis­ti­cal­ly in­signif­i­cant) im­prove­ments in fa­vor of AT-GAA.

Am­i­cus said the FDA is still re­view­ing new­ly sub­mit­ted in­for­ma­tion as part of on­go­ing re­views, but that it is not re­lat­ed to re­quests for ad­di­tion­al clin­i­cal da­ta.

Be­sides, they not­ed, the ad­di­tion­al time should al­low for the agency to in­spect the man­u­fac­tur­ing sites of WuXi Bi­o­log­ics, its con­tract man­u­fac­tur­er, in Chi­na. Un­cer­tain­ty around that in­spec­tion had been a key caveat for ex­ecs who were oth­er­wise up­beat about an OK.

It con­tin­ues to ex­pect reg­u­la­tors to ap­prove the ap­pli­ca­tions to­geth­er, the com­pa­ny added.

So­cial: John Crow­ley, Am­i­cus CEO (via YouTube)

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Todd Zavodnick, Dermavant CEO

With top­i­cal ap­proval, Der­ma­vant looks to bring new stan­dard-of-care to plaque pso­ri­a­sis pa­tients

Dermavant CEO Todd Zavodnick has been plotting to upend the plaque psoriasis market for years now. And with the company’s first approval on Tuesday, he’s pulling the trigger.

The FDA on Tuesday approved Dermavant’s “cosmetically elegant” vanishing cream tapinarof, the first steroid-free topical medication in its class, now marketed as Vtama. And unlike other launches, which could take months, Zavodnick is ready to roll it out as early as next week.

Raymond Tesi, INmune Bio CEO

Man­u­fac­tur­ing con­cerns spur clin­i­cal hold on small biotech's Alzheimer's tri­al — shares plunge

The FDA is keeping an experimental Alzheimer’s therapy out of the US for now, placing a clinical hold on INmune Bio’s IND for a Phase II trial until it can provide additional information on chemistry, manufacturing and controls.

INmune Bio, which gets its name from the approach of leveraging the innate immune system to fight disease, said it was informed about the hold via email and expects more details in a later letter. In a brief statement, it said the agency was looking for more information about CMC “of the newly manufactured XPro1595.”

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