FDA hits Su­per­nus with dou­ble wham­my, hand­ing biotech a CRL and RTF on same day

Su­per­nus had a bad Mon­day.

Yes­ter­day, the Mary­land biotech an­nounced that two dif­fer­ent ther­a­pies had been shot back by the FDA. The agency re­ject­ed their ap­pli­ca­tion to ap­prove SPN-812, an ex­per­i­men­tal AD­HD med­ica­tion for kids and ado­les­cents. And they re­fused to even con­sid­er an ap­pli­ca­tion for SPN-830, a con­tin­u­ous treat­ment pump meant for Parkin­son’s dis­ease.

The re­jec­tion is a sig­nif­i­cant blow to Su­per­nus, which in­vest­ed in four clin­i­cal tri­als for the AD­HD med. The FDA doesn’t dis­close its rea­son­ing for re­ject­ing a drug, but the com­pa­ny said it had to do with CMC is­sues, rather than safe­ty or ef­fi­ca­cy con­cerns. An in-house lab that does an­a­lyt­ic test­ing re­cent­ly changed lo­ca­tions, they said, rais­ing ques­tions in the agency about qual­i­ty con­trol.

Su­per­nus said they plan to “clar­i­fy to the FDA” that they do “not re­ly sole­ly on this fa­cil­i­ty” for qual­i­ty con­trol and then dis­cuss how to re­sub­mit their ap­pli­ca­tion.

The com­pa­ny was less forth­com­ing on why the agency re­fused to file their Parkin­son’s ap­pli­ca­tion. “In the let­ter, the FDA re­quest­ed cer­tain doc­u­ments and re­ports to be sub­mit­ted in sup­port of the ap­pli­ca­tion,” the com­pa­ny said.

Su­per­nus stock $SUPN fell 18%  on the news pre-mar­ket, from 24.95 to 20.35.

Jack Khat­tar

Ex­pec­ta­tions around the AD­HD drug, how­ev­er, had al­ready be­gun to wane. Su­per­nus CEO Jack Khat­tar has billed SPN-812 as the “first tru­ly new ther­a­py” for AD­HD in a decade, but the re­sults in late-stage tri­als fell short of ex­pec­ta­tions.

Al­though pos­i­tive, their first cut of the Phase III study ap­peared to show an ef­fect size no greater than a ri­val Eli Lil­ly drug and failed to show a dose-de­pen­dent re­sponse in the high dose, wor­ry­ing in­vestors. The com­pa­ny has point­ed to da­ta sug­gest­ing their treat­ment can work faster, with pa­tients feel­ing the ef­fect af­ter one week as op­posed to sev­er­al weeks.

The SPN-830 Parkin­son’s pump was part of their $300 mil­lion buy­out of the Ken­tucky-based neu­ro­science com­pa­ny US WorldMeds. Su­per­nus en­vi­sioned it as an al­ter­na­tive to more in­va­sive pro­ce­dures for pa­tients strug­gling with symp­toms, such as the gas­tric tubes some­times used to pro­vide con­tin­u­ous dos­ing of L-Dopa.

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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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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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.