Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

Seiz­ing Aduhelm prece­dent, Bio­gen, Ei­sai get the ball rolling on an­oth­er ac­cel­er­at­ed Alzheimer's ap­proval

Bio­gen — to­geth­er with part­ners at Ei­sai — will be the first to breeze down that Alzheimer’s trail it blazed with the FDA’s his­toric and con­tro­ver­sial ac­cel­er­at­ed ap­proval of Aduhelm.

Ei­sai dis­closed it’s ini­ti­at­ed a rolling BLA sub­mis­sion for lecanemab, or BAN2401, the an­ti-amy­loid be­ta an­ti­body that Bio­gen has long tout­ed as a fol­low-on to Aduhelm (then ad­u­canum­ab). Fol­low­ing the prece­dent that the agency has now set, the two com­pa­nies are gun­ning for an OK based on da­ta sug­gest­ing that the drug could low­er amy­loid be­ta plaques.

They won’t be alone in the queue for long. Roche and Eli Lil­ly are hot on their heels to join the gold rush, with the lat­ter hav­ing al­so pledged to file by the end of the year.

Un­like with Aduhelm, whose ap­proval was ce­ment­ed by mixed da­ta viewed neg­a­tive­ly by the FDA’s ad­vi­so­ry com­mit­tee, the lecanemab fil­ing on­ly comes with Phase IIb da­ta, in­clud­ing those from the open-la­bel ex­ten­sion. The tri­al, dubbed Study 201, en­rolled 856 pa­tients with ear­ly Alzheimer’s and con­firmed amy­loid pathol­o­gy.

“The lecanemab Clar­i­ty AD Phase 3 clin­i­cal tri­al in ear­ly AD is on­go­ing and com­plet­ed en­roll­ment in March 2021 with 1,795 pa­tients,” Ei­sai wrote in a state­ment. “The U.S. FDA has agreed that the re­sults of Clar­i­ty AD, when com­plet­ed, can serve as the con­fir­ma­to­ry study to ver­i­fy the clin­i­cal ben­e­fit of lecanemab. Blind­ed safe­ty da­ta from Clar­i­ty AD will be in­clud­ed to sup­port the BLA.”

A spokesper­son added the pri­ma­ry end­point read­out for Clar­i­ty AD is ex­pect­ed in late 2022.

Dis­cus­sions go at least as far back as Au­gust, when Ei­sai R&D chief Ivan Che­ung told an­a­lysts his team had start­ed talk­ing to the FDA and seek­ing its ad­vice on sub­mit­ting lecanemab to the agency. Weeks be­fore that, the drug was grant­ed break­through ther­a­py des­ig­na­tion by the agency.

If lecanemab does fol­low in the path of Aduhelm and nab an ex­pe­dit­ed green light, it would fur­ther test the lim­its of pay­ers and doc­tors, who have al­ready proven much less wel­com­ing to Aduhelm than reg­u­la­tors (or at least the neu­ro di­vi­sion) were. As End­points News first re­port­ed, the VA de­cid­ed against in­clud­ing the drug in its for­mu­la­ry. Cer­tain pri­vate in­sur­ers have re­port­ed­ly de­clined to cov­er the drug, pend­ing the na­tion­al cov­er­age de­ci­sion by CMS; some treat­ment cen­ters say they won’t ad­min­is­ter the drug, and more re­cent­ly, one cen­ter in Wash­ing­ton, DC even banned Bio­gen rep­re­sen­ta­tives from the premis­es.

ICER, a drug pric­ing watch­dog, added an­oth­er loud voice to the cho­rus by blast­ing Aduhelm’s $56,000 price tag — amid a slate of in­ves­ti­ga­tions where law­mak­ers de­mand­ed an­swers on how the drug was ap­proved.

All of that has trans­lat­ed in­to a painful­ly slow roll­out, which Bio­gen CEO Michel Vounatsos blamed in part on “mis­in­for­ma­tion,” con­fu­sion and neg­a­tive me­dia cov­er­age.

Vounatsos said in the lat­est press re­lease: “It is our vi­sion that pa­tients and their fam­i­lies have choice and ac­cess to mul­ti­ple treat­ment op­tions for Alzheimer’s dis­ease.”

While Aduhelm is de­signed to clear in­sol­u­ble amy­loid plaques, lecanemab most­ly tar­gets sol­u­ble protofib­rils — larg­er oligomers thought to con­tribute to the neu­rode­gen­er­a­tion char­ac­ter­is­tic of Alzheimer’s.

So far, it’s been po­si­tioned for pa­tients with ear­ly Alzheimer’s, mean­ing those with mild cog­ni­tive im­pair­ment due to the dis­ease or mild forms of it.

“We rec­og­nize the strong and ur­gent ex­pec­ta­tions from stake­hold­ers to fur­ther ad­vance a treat­ment sys­tem for this dis­ease,” Ei­sai CEO Haruo Naito said.

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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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.

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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Ted White, Verrica Pharmaceuticals CEO

'Hands may be tied': FDA slaps Ver­ri­ca with 3rd CRL due to prob­lems with con­tract man­u­fac­tur­er

The FDA has rejected Verrica Pharmaceuticals’ skin disease treatment for a third time — and once again the contract manufacturer is to blame.

The biotech emphasized that the only deficiency in the complete response letter is related to a general reinspection of the CMO, Sterling Pharmaceuticals, and has nothing to do specifically with its drug-device; the rest of the NDA is good to go.

CEO Ted White said the company is “extremely disappointed,” but will keep working toward approval.

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