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The FDA granted accelerated approval to Denali Therapeutics’ enzyme replacement therapy for Hunter syndrome, handing a much-needed win to the rare disease market. The drug will carry the brand name Avlayah.

Wednesday’s approval for Avlayah delivers several firsts for the industry. The therapy is the first new medicine for Hunter syndrome in nearly 20 years, Denali said in its press announcement, adding that Avlayah is the first FDA-approved biotherapeutic that uses the transferrin receptor to cross the blood-brain barrier.

Avlayah is also “the first product approved to address neurologic complications of Hunter Syndrome,” according to Tracy Beth Høeg, acting director of FDA’s CDER.

Full approval of Avlayah will hinge on the findings of a confirmatory trial designed to verify its clinical benefits. (Biospace)

The FDA approved Corcept Therapeutics’ Lifyorli in combination with nab-paclitaxel for the treatment of three different kinds of cancer in patients who have received up to three prior lines of treatments.

The March 25 approval came more than three months ahead of the company’s July 11 user fee goal date. In January, the agency rejected the same drug to treat a rare hormonal disorder called Cushing’s syndrome.

A multicenter, open-label trial of 381 patients tested Lifyorli, a selective glucocorticoid receptor antagonist, with nab-paclitaxel for adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer.

It was tested in patients who have received one to three prior lines of treatment, at least one of which included bevacizumab. The results were published in The Lancet last June. (Endpoints)

Rhythm Pharmaceuticals is switching up the tempo for its melanocortin-4 receptor (MC4R) agonist Imcivree.

After its initial approval more than five years ago to treat certain patients with genetic-driven obesity, the drug is moving into a different and broader realm with an FDA nod for acquired hypothalamic obesity (HO).

Acquired HO, for which Imcivree is the first approved treatment, represents an “expanded thinking” on the weight-regulating MC4R pathway that Rhythm’s product targets, CSO Alastair Garfield, Ph.D., said. (Fierce Pharma)

Merck & Co. is spending billions of dollars to bolster its cancer drug portfolio, announcing plans to acquire Terns Pharmaceuticals for a medicine that could disrupt treatment for a type of blood malignancy.

Per deal terms, Merck will pay $53 per share in cash for Terns, an offer that values the company at about $6.7 billion overall.

The deal hands Merck a treatment that has the chance to challenge multiple established medicines in the treatment of chronic myeloid leukemia, a slow-growing cancer of the blood and bone marrow.

The drug, a type of targeted, oral treatment called TERN-701, is currently being studied in an early-stage trial.

Results from that trial, presented at the American Society of Hematology meeting last year, have suggested TERN-701 could threaten the dominance of Novartis’ Scemblix, a medicine expected to generate more than $4 billion in peak yearly sales. (BioPharma Dive)

Breast cancers driven by a particular genetic signature can already be treated by a particular class of targeted therapies, but the race is on to develop next-generation medicines that improve the safety and efficacy of this approach.

Novartis sees an early clinical program of Synnovation Therapeutics as potentially best in this drug class, and it is paying $2 billion to add the clinical-stage asset to its pipeline.

The cancer drug comes from Pikavation Therapeutics, a subsidiary of precision medicine startup Synnovation. By acquiring Pikavation, Novartis gets that subsidiary’s entire portfolio of PI3Kα inhibitors, according to deal terms announced on March 20.

Ionis Pharmaceuticals is dramatically lowering the price of its blockbuster hopeful Tryngolza (olezarsen) as it prepares to grow the medicine’s reach with a planned label expansion.

Initially approved as a treatment for familial chylomicronemia syndrome (FCS) in 2024, the drug first launched with an annual list price of $595,000, the company said.

Now, as Ionis gears up to potentially launch in severe hypertriglyceridemia (sHTG), the company is rolling out an updated wholesale acquisition cost (WAC) of $40,000 annually, it announced.

Tryngolza’s new price will take effect April 1 and will apply to both the anticipated sHTG indication and its current use in FCS.

Not only does the sharp WAC decrease support broader access and speak to the higher cost of drugs in the rare disease realm, but it could also give Ionis a better shot against its market rival, Arrowhead’s Redemplo. (Fierce Pharma)

While the company plans to move forward with the FDA approval process, the vaccine fell short of their goal to demonstrate 20 percent effectiveness in worst-case scenarios.

If it manages to succeed, it would be the only Lyme disease vaccine approved for humans.

GSK cited insufficient demand when withdrawing it from the market in 2002, but what really happened has been called a “cautionary tale” for vaccine developers.