Drug Approvals

May 9: The FDA approved Chiesi Group unit Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc.’s Elfabrio (pegunigalsidase alfa-iwxj) for the treatment of adults with Fabry disease. A PEGylated enzyme replacement therapy, the agent is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life. Recommended dosing is 1 mg/kg via intravenous infusion every two weeks.

May 17: The FDA approved a 40 mg/0.8 mL single-dose autoinjector for Boehringer Ingelheim’s Cyltezo (adalimumab-adbm). The agency initially approved the biosimilar of AbbVie Inc.’s Humira (adalimumab) on Aug. 25, 2017, as a single-use prefilled syringe. The drug is approved for multiple chronic inflammatory diseases. The new pen will be available in two-, four- and six-pack options when the tumor necrosis factor (TNF) inhibitor launches on July 1.

In recent months, the interest in prescription weight loss medications has grown exponentially as celebrities tout the drugs and clinical trial results show their safety and effectiveness. However, while tens of millions of people could benefit from the medications, health policy experts tell AIS Health that payers are taking a cautious approach and are concerned at the financial impact if the FDA approves more drugs for that indication, as is expected.

A recent survey from the International Foundation of Employee Benefit Plans found that 22.1% of employers last year offered prescription drug coverage for weight loss, including 21.3% of corporations, 22% of public employers and 27% of multiemployer plans. Of the 470 companies that responded to the survey, 25% said obesity is one of the top three health conditions that has the largest impact on overall health costs.

In recent months, the interest in prescription weight loss medications has grown exponentially as celebrities tout the drugs and clinical trial results show their safety and effectiveness. However, while tens of millions of people could benefit from the medications, health policy experts tell AIS Health that payers are taking a cautious approach and are concerned at the financial impact if the FDA approves more drugs for that indication, as is expected.

A recent survey from the International Foundation of Employee Benefit Plans found that 22.1% of employers last year offered prescription drug coverage for weight loss, including 21.3% of corporations, 22% of public employers and 27% of multiemployer plans. Of the 470 companies that responded to the survey, 25% said obesity is one of the top three health conditions that has the largest impact on overall health costs.

Eisai Co., Ltd. and Biogen Inc.’s Leqembi (lecanemab) could raise Medicare Part B spending by $8.9 billion or more annually if it receives full approval from the FDA KFF found. And a report by researchers from the RAND Corp. found that Leqembi could increase spending by $2 billion to $5 billion. KFF’s projection was based on an annual list price of $26,500 and uptake among 5% of Alzheimer’s patients. The RAND report included estimates of ancillary costs, such as MRI scans. The FDA granted accelerated approval to the anti-amyloid monoclonal antibody treatment in January, and its decision on full approval is expected soon.

CVS Health Corp is shutting down its clinical trials division, which launched in 2021, and will conclude operations in 2024. The division has worked with more than 30 drugmakers on 50 Phase I, II and IV studies involving 33,000 participants, according to Modern Healthcare.

As CMS continues to tighten oversight of misleading marketing activities, the agency will soon require Medicare Advantage organizations to file all materials that mention any type of benefit. In a May 10 memo from the Medicare Drug & Health Plan Contract Administration Group, CMS explained that while it previously interpreted the mentioning of widely available benefits (e.g., vision, dental, premium reductions) as “general descriptions” that were not “made with sufficient intent to draw attention to a particular plan or subset of plans” and lead to an enrollment decision “without information on the associated costs for enrollees,” complaints received through various channels have indicated otherwise. Therefore, CMS is expanding its interpretation of “marketing” to “include content that mentions any type of benefit covered by the plan and is intended to draw a beneficiary’s attention to plan or plans, influence a beneficiary’s decision-making process when selecting a plan, or influence a beneficiary’s decision to stay enrolled in a plan (that is, retention-based marketing) and thus subject to review.” As such, the agency will require any material or activity that is distributed by any means and mentions any benefit to be submitted into the Health Plan Management System effective July 10.

GlaxoSmithKline Pharmaceuticals Ltd. won the first US approval for a respiratory syncytial virus vaccine on May 3 in older adults but looks like it may not be able to claim the same crown in the other key population impacted by the virus — infants and young children.

The Food and Drug Administration approval letter for GSK’s Arexvy says the agency is waiving the pediatric study requirement for children ages 0 to 2 “because there is evidence strongly suggesting that the biological product would be unsafe in this pediatric group.”

The label for Arexvy, which is indicated for adults 60 and older, says “evidence from an animal model strongly suggests that Arexvy would be unsafe in individuals younger than 2 years of age because of increased risk of enhanced respiratory disease.”

April 11: The FDA expanded the patient population of Takeda’s HyQvia (immune globulin infusion 10% [human] with recombinant human hyaluronidase) to include the treatment of primary immunodeficiency in people between the ages of 2 to 16 years. The agency first approved the drug on Sept. 12, 2014. Subcutaneous dosing can be administered at home or in an infusion center every three or four weeks after a ramping-up period. The volume administered is based on weight and trough level. Drugs.com lists the price of 160 u/mL for 105 milliliters as more than $2,464.

April 14: The FDA expanded the label of Novartis Pharmaceutical Corp. unit Sandoz Inc.’s Hyrimoz (adalimumab-adaz) for the treatment of moderate-to-severe hidradenitis suppurativa in adults. The agency initially approved the biosimilar of AbbVie Inc.’s Humira (adalimumab) on Oct. 30, 2018. Dosing starts with 160 mg via subcutaneous injection on day one or split over two consecutive days, then 80 mg on day 15, and then on day 29 and subsequent doses, 40 mg every week or 80 mg every other week. Drugs.com lists the price of two 40 mg/0.4 mL kits and two 40 mg/0.8 mL kits of Humira, each with two devices, as more than $7,299. Sandoz has said it will launch Hyrimoz on July 1 per the settlement of patent litigation with AbbVie.

Oklahoma lawmakers accused CVS Health Corp.’s Caremark PBM of “intentionally lying” in its denials of 90-day prescription fills for some patients, according to The Oklahoman. The Oklahoma City newspaper reported that Caremark had routinely denied Oklahoma residents 90-day prescription fills and cited a state law as the reason, claiming that such fills were illegal under the statute. State Rep. Jon Echols, a Republican, said that such a justification was a lie: “Anyone that says that is not misinformed. They don’t misunderstand. They are intentionally lying to you. And we’re not going to stand for it.” Oklahoma’s insurance commissioner, Republican Glen Mulready, earlier this month filed an administrative action against Caremark that, per a press release from Mulready’s office, “targets the practice of ‘steering patients’ to CVS pharmacies and prescription mail-order services and seeks to censure, suspend, place on probation or revoke the Pharmacy Benefit Manager (PBM) license of CVS/Caremark. In addition, the… Department will seek restitution and/or levy fines for each alleged violation.”

The FDA recently approved a new first-in-class agent for follicular lymphoma, an important development for people with the condition, according to one oncologist. Payers and oncologists both agreed that the drug’s approval should help ease the disease burden somewhat for people suffering from the condition, according to a Zitter Insights survey.

On Dec. 22, the FDA gave accelerated approval to Roche Group member Genentech USA, Inc.’s Lunsumio (mosunetuzumab-axgb) for the treatment of adults with relapsed or refractory follicular lymphoma after at least two lines of systemic therapy. The drug is a first-in-class CD20xCD3 T-cell engaging bispecific antibody. Dosing for the agent — which can be done in an outpatient setting — is 1 mg via intravenous infusion on day one of cycle one, 2 mg on day eight of cycle one and 60 mg on day 15 of cycle one, each over a minimum of four hours. On day one of cycle two, dosing is 60 mg, and then in cycle three and following cycles, dosing is 30 mg on day one; administration can be reduced to two hours if cycle one infusions were tolerated. Dosing for eight cycles is recommended. If people have a partial response or stable disease after eight cycles, an additional nine cycles may be administered. The drug’s price for eight cycles is about $180,000.

March 10: The FDA approved Acadia Pharmaceuticals Inc.’s Daybue (trofinetide) for the treatment of Rett syndrome in people at least 2 years old. The drug is the only FDA-approved treatment for the complex, rare neurodevelopmental disorder, which is estimated to affect 6,000 to 9,000 people in the U.S. The agency gave the drug fast track status and orphan drug designation; it also gave the company a rare pediatric disease priority review voucher. Dosing of the oral solution is weight based and can be given orally or via gastrostomy tube. The company has not disclosed the agent’s price, but analyst estimates are between $400,000 and $600,000 per year. Acadia said it expects the agent to be available by the end of April.

March 13: The FDA expanded the patient population of Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) to include the treatment of cholestatic pruritus in people with Alagille syndrome who are at least 3 months old. The agency initially approved the oral solution on Sept. 29, 2021. The starting dose is 190 mcg/kg once daily and then increased to 380 mcg/kg after one week. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters as more than $53,712.