Atsushi “Andy” Fujimoto, Mitsubishi Tanabe Pharma America president

FDA ap­proves oral for­mu­la­tion of old ALS drug, giv­ing pa­tients new treat­ment op­tion in sore­ly need­ed field

Much of the ALS drug news in re­cent months has fo­cused on Amy­lyx Phar­ma­ceu­ti­cals and its ex­per­i­men­tal pro­gram re­viewed by an FDA ad­vi­so­ry com­mit­tee in March. But on Thurs­day, the FDA green­light­ed a new way to take an al­ready-ap­proved ALS drug that flew un­der the radar.

US reg­u­la­tors OK’ed an oral for­mu­la­tion of Rad­i­ca­va, first ap­proved to treat ALS back in 2017 as a se­ries of IV in­fu­sions, to treat adults with ALS, the agency an­nounced Thurs­day af­ter­noon. The de­ci­sion gives pa­tients an­oth­er op­tion for a uni­ver­sal­ly fa­tal dis­ease with on­ly one oth­er ap­proved drug.

FDA grant­ed the ap­proval to Mit­subishi Tan­abe Phar­ma Amer­i­ca, which sub­mit­ted for the ap­proval in Jan­u­ary and was grant­ed pri­or­i­ty re­view.

ALS is a dev­as­tat­ing dis­ease, with most pa­tients dy­ing be­tween three and five years af­ter the on­set of symp­toms. New treat­ments are des­per­ate­ly need­ed, as Rad­i­ca­va and rilu­zole, ap­proved in the mid-1990s, re­main the on­ly two ap­proved drugs.

Where­as rilu­zole can be tak­en oral­ly, Rad­i­ca­va (edar­avone) pre­vi­ous­ly need­ed to be ad­min­is­tered through sev­er­al IV in­fu­sions. Pa­tients need­ed to at­tend clin­ics or spe­cial ALS sites to re­ceive the hour-long in­fu­sions for 14 straight days in the first 28-day cy­cle, and then in 10 out of 14 days for every cy­cle there­after.

Now, pa­tients can take the oral for­mu­la­tion at home them­selves, or through a feed­ing tube. The ad­min­is­tra­tion sched­ule re­mains the same, the FDA said, but pa­tients must al­so now fast overnight be­fore tak­ing the oral ver­sion.

Rad­i­ca­va was grant­ed its first ap­proval af­ter be­ing able to show a mod­est ben­e­fit in slow­ing func­tion­al de­cline, us­ing a clin­i­cal­ly val­i­dat­ed yet in­creas­ing­ly con­tro­ver­sial mea­sure­ment. Mit­subishi Tan­abe Phar­ma earned the new OK thanks to a study show­ing com­pa­ra­ble lev­els of the drug in pa­tients’ blood­streams to that seen from the IV for­mu­la­tion.

Thurs­day’s de­ci­sion comes as pa­tients have been ramp­ing up the pres­sure on reg­u­la­tors in the last few years, ar­gu­ing the re­view process is too slow. The pa­tient com­mu­ni­ty notched a win back in 2019 when the FDA up­dat­ed its guid­ance for de­vel­op­ing new ALS drugs, in­clud­ing a pro­vi­sion ad­vis­ing against deny­ing ap­proved treat­ments in place­bo arms.

More re­cent­ly, pa­tients have ral­lied around the Amy­lyx drug in the hopes of sway­ing the FDA’s ex­perts. At the Amy­lyx ad­comm ear­li­er this year, rough­ly 30 pa­tients spoke dur­ing the near­ly two-hour pub­lic com­ment pe­ri­od, stress­ing the need for new ALS drug ap­provals.

It re­mains to be seen whether reg­u­la­tors will green­light the Amy­lyx pro­gram, dubbed AMX0035. Though the drug achieved sta­tis­ti­cal sig­nif­i­cance in a Phase II study mea­sur­ing func­tion and ap­peared to show some sur­vival ben­e­fit in a long-term fol­low-up, out­stand­ing ques­tions re­mained over the qual­i­ty of the lat­ter dataset.

The FDA has been more open to re­view­ing the Amy­lyx drug, how­ev­er. Af­ter ini­tial­ly re­quest­ing the biotech con­duct a large-scale Phase III tri­al be­fore sub­mit­ting its NDA, reg­u­la­tors changed their minds and al­lowed the sub­mis­sion based on just the Phase II and long-term stud­ies.

FDA’s ad­vi­sors rec­om­mend­ed in a close 6-4 vote against ap­proval us­ing just the cur­rent­ly avail­able da­ta. The agency is ex­pect­ed to make a de­ci­sion by June 29.

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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