FDA

‘Buckle Up’: Second Trump Administration May Be ‘Mixed Bag’ for Health Care, Biotech

The second administration of Donald Trump may well run the gamut as far as its impact on health care and pharma. Biotech companies may benefit from a good business environment, prompting more mergers and acquisitions, but they may also experience challenges in working with what could be somewhat unconventional leaders of federal agencies, such as Robert F. Kennedy Jr., whom President-elect Trump tapped on Nov. 14 to run HHS.

RFK Jr., a politician and environmental activist who has questioned the safety and efficacy of vaccines and spread misinformation about them, has said that “there are entire departments, like the nutrition department, at FDA that have to go, that are not doing their job.”

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First Dual Inhibitor of Its Kind Is Expected to Have Some Impact in Psoriatic Arthritis

A recently approved treatment for psoriatic arthritis (PsA) brings a new mechanism of action to the therapeutic class. And while payers and rheumatologists varied in their expectations of what the drug’s impact on coverage of and prescribing for the class will be, almost half said they expect it to have at least some effect, according to a survey by Zitter Insights.

On Sept. 20, the FDA approved three more indications for UCB, Inc.’s Bimzelx (bimekizumab-bkzx) for the treatment of adults with active PsA, adults with active non-radiographic axial spondyloarthritis with objective signs of inflammation and adults with active ankylosing spondylitis. Two months later, on Nov. 20, the FDA gave the drug another approval, for the treatment of moderate-to severe hidradenitis suppurativa.

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Lilly’s Ebglyss Brings Another IL-13 Inhibitor to Atopic Dermatitis Treatment

Last month’s FDA approval of a drug to treat atopic dermatitis brings a new agent to a growing class of medications that payers say they consider a high priority to manage.

On Sept. 13, the FDA approved Lilly’s Ebglyss (lebrikizumab-lbkz) for the treatment of people at least 12 years old who weigh at least 40 kg with moderate-to-severe atopic dermatitis that is not well controlled despite treatment with topical prescription medications or when those medications are not advisable. Dosing for the interleukin-13 (IL-13) antagonist is 500 mg via two 250 mg subcutaneous injections at weeks zero and two and then 250 mg every two weeks until week 16 or later, when an adequate clinical response is achieved. At that point, maintenance dosing is 250 mg every four weeks. The drug’s WAC is $3,500 for a 250 mg/2 mL single-dose prefilled pen or prefilled syringe.

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Comments on FDA Interchangeability Draft Guidance Run the Gamut

Over the last few years, the FDA has taken multiple steps to level the playing field between biosimilars and interchangeable biosimilars. More recently, it proposed draft guidance that would do away with switching studies for interchangeability status. Commenters on that guidance were mostly supportive — with some even backing interchangeability for all biosimilars — and others asked for clarification on a range of issues, including what information the FDA needed to make a determination of interchangeability. Meanwhile, one group derided the guidance as an “ill-advised and inappropriate move.”

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New FDA Approvals: FDA Approved Citius’ Lymphir for CTCL

Aug. 7: The FDA approved Citius Pharmaceuticals, Inc.’s Lymphir (denileukin diftitox-cxdl) for the treatment of relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one systemic therapy. The immunotherapy is the only CTCL treatment that targets the interleukin-2 (IL-2) receptor found in T-cell lymphomas and regulatory T cells (Tregs). The rare disease is a chronic non-Hodkin lymphoma that primarily affects the skin. The drug is a reformulation of Ontak (denileukin diftitox), which was on the U.S. market from 2008 to 2014, when it was voluntarily withdrawn “to enable manufacturing improvements.” The FDA gave the therapy orphan drug designation. The recommended dose is 9 mcg/kg per day based on actual body weight via a 60-minute intravenous infusion on days one through five of a 21-day cycle. The company says it expects to launch the agent within the next five months.

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As Biden Admin Winds Down, Will It Address Accumulators, Maximizers as Promised?

As President Joe Biden’s administration nears its end, two promised rules on copayment accumulators and maximizers have yet to be released. They stand to have a huge impact on whether pharma manufacturer-provided patient assistance — much of which is provided for specialty drugs — must be counted toward patients’ out-of-pocket responsibility.

The first concerns a lawsuit over the 2021 Notice of Benefit and Payment Parameters (NBPP) and its stance toward copay accumulators.

Health plans and PBMs several years ago began implementing accumulators to counter manufacturer copay assistance programs. Traditionally, that assistance would count toward beneficiaries’ annual out-of-pocket expenses. When those out-of-pocket maximums were reached, health plans would cover the remainder of members’ costs for the year. With accumulators, patients can still use that assistance, but it does not help reduce their out-of-pocket costs.

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Yimmugo May Help Ease ‘Shortage Stress,’ Promote Competition

A newly approved immune globulin for primary immunodeficiency will bring another option to a class of products that often is plagued by shortages. It is also a crowded space, and payers have several considerations when deciding on the coverage of these agents, say industry experts.

On June 13, the FDA approved Grifols Group company Biotest AG’s Yimmugo (immune globulin intravenous, human-dira) for the treatment of primary humoral immunodeficiency in people at least 2 years old. Dosing via intravenous infusion is every three to four weeks, with the first infusion started at 0.5 mg/kg per minute and increasing up to 3.0 mg/kg per minute; afterwards, the maintenance infusion rate is 13 mg/kg per minute. The dosage can be adjusted over time to achieve the desired trough levels and clinical response.

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In Push for More Real-World Evidence, Payers Can Play Critical Role

Commercial and government payers can play a key role in generating real-world evidence (RWE) and helping the health care system reduce costs and improve outcomes, according to a special communication published last month in JAMA. Meanwhile, speakers at a July 25 virtual meeting sponsored by Duke University’s Margolis Institute for Health Policy emphasized that payers should work together with pharmaceutical companies, medical device manufacturers and policymakers to institute better ways to incorporate RWE into practice.

Mark McClellan, M.D., Ph.D., director of the Margolis Institute and a former FDA commissioner and HHS administrator, defined real world data as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources” such as registries, wearable devices and electronic health records. He added that RWE is “clinical evidence about the use, potential benefits or risks of a medical product or practice or care delivery model that’s derived from the analysis of real-world data.”

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New FDA Approvals: FDA Approved ANI’s Generic Endari

July 8: The FDA approved ANI Pharmaceuticals, Inc.’s L-glutamine oral powder to reduce the acute complications of sickle cell disease in people at least 5 years old. The company says it is the first AA-rated approved generic for Emmaus Medical, Inc.’s Endari. Dosing for people weighing less than 30 kilograms is one packet twice daily; for those between 30 and 65 kilograms, dosing is two packets twice daily, and for those more than 65 kilograms, dosing is three packets twice daily. Drugs.com lists the price of 60 packets of Endari as more than $1,505.

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Commercial Plans Are Likely to Approach New Alzheimer’s Drug With Caution

Although the approval of Kisunla (donanemab) earlier this month was celebrated by the Alzheimer’s Association as “real progress,” the challenge of getting commercial insurers to cover the drug will likely end up tempering that excitement.

Eli Lilly’s Kisunla, which received full FDA approval on July 2, is a once-monthly, IV-infused treatment for people with early symptoms of Alzheimer’s disease, including mild cognitive impairment and mild dementia. Like the drug Leqembi (lecanemab), which received full approval last July, Kisunla aims to slow the progression of Alzheimer’s by removing amyloid plaques from the brain. (The naturally occurring protein amyloid can create plaques when clumped together, and if those plaques build up excessively in the brain, it may lead to memory and cognitive issues associated with Alzheimer's disease.)

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