That big new hy­per­parathy­roidism drug from Am­gen? The FDA re­ject­ed it

Ac­cord­ing to reg­u­lar up­dates from Am­gen over the past two years, in­ves­ti­ga­tors marched through a trio of late-stage stud­ies for etel­cal­ce­tide (AMG416) with noth­ing but good things to re­port. The drug scored out­stand­ing da­ta for sec­ondary hy­per­parathy­roidism (SH­PT), a dis­ease that af­fects chron­ic kid­ney dis­ease pa­tients on he­modi­a­lyis.

And this evening Am­gen re­port­ed that the drug was re­ject­ed by the FDA. The news came in a terse state­ment, with­out a hint about what the agency ob­ject­ed to.

The com­mer­cial name for this drug is Parsabiv. And here’s what Am­gen — a big biotech not known for ex­plain­ing dis­taste­ful things — had to say about it:

Am­gen is re­view­ing the Com­plete Re­sponse Let­ter, and we an­tic­i­pate a post-ac­tion meet­ing with the FDA lat­er this year to dis­cuss the Com­plete Re­sponse. The Com­plete Re­sponse Let­ter does not im­pact our reg­u­la­to­ry sub­mis­sions in oth­er re­gions.

Am­gen test­ed the drug in more than a thou­sand pa­tients, re­port­ed­ly meet­ing its pri­ma­ry end­points by slash­ing dan­ger­ous­ly high parathy­roid hor­mone lev­els. In one study 74.7% of sub­jects on the drug hit its tar­get, com­pared to just 8.9% in the place­bo arm. In an­oth­er Phase III tri­al the new drug out­per­formed Am­gen’s block­buster drug Sen­si­par, which earned $1.4 bil­lion.

This was one of Am­gen’s top Phase III ef­forts in the pipeline. It scored a suc­cess on its PC­SK9 cho­les­terol drug, but it’s been a dis­ap­point­ment in its ear­ly days on the mar­ket.

Bri­an Sko­r­ney, Baird an­a­lyst

Baird’s Bri­an Sko­r­ney not­ed the ab­sence of any ex­pla­na­tion from Am­gen, and then of­fered his opin­ion that the agency might have been put off by safe­ty is­sues. His com­ment:

In the head-to-head study, Sen­si­par and Parsabiv looked sim­i­lar, with Parsabiv show­ing high­er rates of treat­ment-emer­gent AEs (93% vs. 90%) over­all, with a slight­ly low­er rate of se­ri­ous ad­verse events (25% vs. 27%). Specif­i­cal­ly, Parsabiv showed high­er in­ci­dence of hypocal­cemia (5% vs. 2.3%), car­diac fail­ure (3% vs. 0.6%), and fa­tal ad­verse events (2.7% vs. 1.8%). It re­mains un­clear whether or not these dif­fer­ences fac­tored in­to the FDA’s de­ci­sion. It is pos­si­ble that the mod­est in­crease in these ad­verse events out­weighed the ben­e­fits pro­vid­ed on sec­ondary end­points and in the dif­fer­ing ad­min­is­tra­tion, in the FDA’s opin­ion. It’s al­so pos­si­ble that this is just a mi­nor CMC is­sue and can be quick­ly re­solved.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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