So­bi's high-pri­or­i­ty rare dis­ease drug runs in­to a wall at EMA

The EMA has re­buffed Swe­den-based So­bi’s pitch for ema­palum­ab, shoot­ing down high hopes for a $568 mil­lion drug.

So­bi said it will re­quest a re-ex­am­i­na­tion by the reg­u­la­tors, which will kick off a new re­view process that should pro­duce a de­ci­sion by the end of the year. The an­ti­body is de­signed to treat pri­ma­ry he­mo­phago­cyt­ic lym­pho­his­ti­o­cy­to­sis in chil­dren un­der 18 years old by tar­get­ing in­ter­fer­on gam­ma (IFNγ).

The drug, al­ready ap­proved in the US as Gam­i­fant, is one of the stars of So­bi’s pipeline af­ter it re­struc­tured its op­er­a­tions to fo­cus on hema­tol­ogy and im­munol­o­gy.

The com­pa­ny first li­censed it from Swiss biotech Novim­mune in 2018, pay­ing $50 mil­lion and promis­ing $400 mil­lion in mile­stones be­fore de­cid­ing to ac­quire the whole thing — com­plete with in­tel­lec­tu­al prop­er­ty rights, the team work­ing on it and a pri­or­i­ty re­view vouch­er. Un­der the new deal, mile­stones add up to $518 mil­lion.

“Dur­ing the last years our team has gained a lot of ex­pe­ri­ence in this rather com­plex dis­ease area,” CEO Gui­do Oelk­ers said in a state­ment, as over 100 pa­tients have been treat­ed in the US.

He didn’t elab­o­rate on what might have gone wrong in the ap­pli­ca­tion. Fran­co Lo­catel­li, the prin­ci­pal in­ves­ti­ga­tor in the EU, was quot­ed say­ing he was “sig­nif­i­cant­ly sur­prised” about the EMA rec­om­men­da­tion against ap­proval.

“I had the priv­i­lege to ob­serve that this mon­o­clon­al an­ti­body, tar­get­ing the main cy­tokine in­volved in the dis­ease patho­phys­i­ol­o­gy, was well tol­er­at­ed and ef­fec­tive in a large pro­por­tion of the pa­tients, rep­re­sent­ing a mod­el of pre­ci­sion med­i­cine,” he said. “While US chil­dren have since al­most 2 years the pos­si­bil­i­ty to be treat­ed with this nov­el, safe, high­ly ef­fec­tive and tar­get­ed ther­a­py, the EMA de­ci­sion paves the way for mi­gra­to­ry health flows to­wards non-Eu­ro­pean Cen­ters that can grant this treat­ment.”

In the re­lease So­bi al­so re­it­er­at­ed its es­ti­mat­ed peak sales tar­get of over $500 mil­lion — which “re­mains un­changed re­gard­less of an ap­proval in Eu­rope.”

Sales of Gam­i­fant in the first six months of 2020 were $26.7 mil­lion. In 2019, the first year of launch, to­tal sales were $61.3 mil­lion.

By So­bi’s es­ti­mate, the most im­por­tant mar­kets based on num­ber of pa­tients for HLH — a rare but se­vere dis­ease that can re­sult in fever, en­large­ment of the liv­er and spleen, en­larged lymph nodes, rash­es and hos­pi­tal­iza­tions — are Chi­na, fol­lowed by the US, Eu­rope and Japan.

It’s al­so ini­ti­at­ing clin­i­cal stud­ies for pre-emp­tive treat­ment of pa­tients with risk fac­tors of HSCT acute graft fail­ure.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

Kirk Myers is shown in a still image from a new film series showcasing the efforts of HIV advocates funded by Gilead.

Gilead spot­lights HIV projects and the com­mu­ni­ty lead­ers dri­ving them in new mi­ni-doc­u­men­tary films

Gilead is going behind the scenes of some of the HIV initiatives it funds through grants in a new film series narrated by the people helming the projects.

The first four films and leaders come from across the US — Arianna Lint in Florida and Puerto Rico, Cleve Jones in San Francisco, June Gipson in Mississippi and Kirk Myers in Texas. Their HIV-focused efforts range from addressing unmet needs of the transgender community to delivering social services and high-quality health care in underserved communities.

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EMA pulls an opi­oid from the 1950s used to treat dry cough

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

FDA's drug short­ages leader wants com­pa­nies to start re­port­ing in­creas­es in de­mand

It is no secret that drug shortages have been prevalent in 2022. Several major drug products, such as amoxicillin and Adderall, have been in short supply for several months and have led to members of Congress applying pressure on the FDA and HHS to resolve the situation.

Speaking at a webinar hosted by the Alliance for a Stronger FDA, Valerie Jensen, the associate director of the FDA’s Drug Shortage Staff, noted both the rise in quality-related issues and increased demand for some products. She called on companies to report such demand increases, even though they are not currently required to do so.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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