FDA

The FDA recently approved the first biosimilar for the treatment of multiple sclerosis (MS): Tyruko (natalizumab-sztn) from Novartis Pharmaceutical Corp. subsidiary Sandoz Inc. The drug is entering a fairly crowded class, and payers have said they expect it to have a moderate impact on their management of the other agents available to treat the condition.

On Aug. 24, the FDA approved Tyruko for the treatment of two indications: (1) adults with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease, and (2) adults with moderately to severely active Crohn’s disease with evidence of inflammation who have had an inadequate response to, or are unable to tolerate, conventional Crohn’s therapies and tumor necrosis factor (TNF) inhibitors.

The Center for Medicare Advocacy and the Community Legal Services clinic at the University of the Pacific, McGeorge School of Law filed a class action lawsuit (Case 2:23-cv-01932-DB) against HHS on Sept. 8 on behalf of Medicare beneficiaries who have lost coverage of certain Medicare Part B drugs. Plaintiffs were receiving the Janssen Pharmaceutical Companies of Johnson & Johnson’s Stelara (ustekinumab) in an outpatient clinic, where the agent was administered by health care professionals and covered under Part B as an agent provided “incident to” a physician’s service due to disabilities that prevent them from self-administration. In October 2021, HHS denied coverage of Stelara under Part B because it had determined that the drug is “usually self-administered by the patient.” HHS did not notify patients about the change, and it did not require providers to issue a notice. One plaintiff had four injections of more than $40,000 each, while another had two injections of about $58,000 each, before they realized that they were responsible for the full cost of the drug. Plaintiffs are seeking “timely, adequate notice” be required when a Part B drug that has been furnished incident to a practitioner’s service is added to the self-administered drug (SAD) list, as well as a modification to Medicare allowing beneficiaries who cannot self-administer a medically necessary drug due to a disability to be able to receive Medicare-covered medications administered by a health care professional, among other requests.

Aug. 9: The FDA gave accelerated approval to the Janssen Pharmaceutical Companies of Johnson & Johnson’s Talvey (talquetamab-tgvs) for the treatment of adults with relapsed or refractory multiple myeloma who have received at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody. The review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration and Switzerland’s Swissmedic; it used the Assessment Aid. The agent, a G protein-coupled receptor class C group 5 member D (GPRC5D)-directed CD3 T-cell engager, is a first-in-class bispecific antibody. Dosing of the subcutaneous injection, which is administered by a qualified health care professional, can be done weekly or biweekly. For weekly dosing, a step-up schedule consists of 0.01 mg/kg on day one, then 0.06 mg/kg on day four, then 0.4 mg/kg on day seven and then 0.4 mg/kg one week later and weekly thereafter. For biweekly dosing, administration starts at 0.01 mg/kg on day one, 0.06 mg/kg on day four, 0.4 mg/kg on day seven, then 0.8 mg/kg on day 10 and then 0.8 mg/kg two weeks later and every two weeks thereafter. The drug’s list price is $45,000 per month, and the company estimates a pricing range of $270,000 to $360,000 for an average treatment duration of six to eight months.

The FDA recently granted another approval to Taiho Pharmaceutical Co., Ltd. division Taiho Oncology, Inc.’s Lonsurf (trifluridine/tipiracil) for a type of colorectal cancer. The decision provides another treatment option for a condition that respondents to a Zitter Insights survey regard as in need of more effective therapies.

On Aug. 2, the FDA approved Lonsurf as a single agent or in combination with bevacizumab for the treatment of adults with metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-vascular endothelial growth factor (VEGF) drug and, if Rat sarcoma (RAS) wild-type, an anti-epidermal growth factor receptor (EGFR) therapy. The agency first approved the oral nucleoside antitumor agent on Sept. 22, 2015. The newest use had priority review. Dosing for the tablet is 35 mg/m2 twice daily on days one through five and days eight through 12 of each 28-day cycle. Drugs.com lists the price of 20 6.14 mg/15 mg tablets as more than $4,204.

Sen. Mike Lee (R-Utah) reintroduced the Biosimilar Red Tape Elimination Act (S. 2305), his office said on July 13. The legislation is focused on increasing competition among biologics and lowering consumer costs for them. He first introduced the bill, which would do away with the FDA requirement for switching studies for biosimilars seeking the interchangeability designation, on Nov. 17, 2022. The legislation has been referred to the Senate Committee on Health, Education, Labor, and Pensions.

Clearway Health is working with The Brooklyn Hospital Center to help improve access to specialty pharmacy drugs for underserved patients, Clearway Health said on Aug. 2. The company, which partners with hospitals and health systems to build or improve their own specialty pharmacy programs, said it will help the hospital by broadening its services, decreasing patients’ financial responsibilities, improving patient adherence and boosting clinical outcomes.

July 7: The FDA broadened the label of Novartis Pharmaceuticals Corp.’s Leqvio (inclisiran) to include its use as an adjunct to diet and statin therapy to reduce low-density lipoprotein cholesterol (LDL-C) in adults with primary hyperlipidemia. The agency initially approved the small interfering RNA (siRNA) therapy on Dec. 22, 2021. Dosing is 284 mg via a subcutaneous injection by a health care professional, then another dose at three months and then every six months. The drug’s list price is $3,290.63 per dose.

July 11: The FDA expanded the label of Organon’s Hadlima (adalimumab-bwwd) to include the treatment of adults with non-infectious intermediate and posterior uveitis and panuveitis. The agency first approved the biosimilar of AbbVie Inc.’s Humira (adalimumab) on July 23, 2019. Dosing starts with 80 mg via subcutaneous injection, followed by 40 mg every other week starting one week after the initial dose. The agent is available in both low-concentration and high-concentration versions of its reference drug. The product recently launched with a wholesale acquisition cost of $1,038 for a carton of two syringes or autoinjectors.

Demand for Novo Nordisk A/S’s Wegovy (semaglutide) may spike even higher following the Danish pharma giant’s Aug. 8 announcement that the diabetes drug, recently approved by the FDA for use as a weight loss treatment, “reduces the risk of major adverse cardiovascular events by 20%” in patients with obesity. Clinicians and pharmacists say the drug’s broad appeal became even greater with the news but suggest that insurers and plan sponsors are likely to continue to erect access barriers to Wegovy given its high cost and clinical limitations.

Novo said a double-blinded trial “compared subcutaneous once-weekly semaglutide 2.4 mg with placebo as an adjunct to standard of care for prevention of major adverse cardiovascular events (MACEs) over a period of up to five years,” and “achieved its primary objective by demonstrating a statistically significant and superior reduction in MACE of 20% for people treated with semaglutide 2.4 mg compared to placebo.”

In its latest drug pipeline insights report, Optum Rx pivots from its habit of highlighting upcoming medications for rare diseases, instead focusing on a trio of emerging therapies that target much wider patient populations. The UnitedHealth Group-owned PBM tells AIS Health, a division of MMIT, that the move was driven more by happenstance than intention.

“In this report, we were focusing on drugs expected in the third quarter of 2023, and from among that group, these three seemed particularly interesting to describe in more detail,” explains Bill Dreitlein, senior director of pipeline and drug surveillance at Optum Rx.

The FDA recently converted accelerated approval to full for Amgen Inc.’s Blincyto (blinatumomab) for certain patients with acute lymphoblastic leukemia (ALL). While the drug is one of many others approved for the condition, respondents to a Zitter Insights survey said there is still unmet need in treating the disease.

On June 21, 2023, the FDA granted full approval to Blincyto for the treatment of adults and pediatric patients with CD19-positive B-cell precursor ALL — which is also known as acute lymphocytic leukemia — in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%. The agency first approved the CD19-directed CD3 T-cell engager on Dec. 3, 2014; the accelerated approval for MRD-positive B-cell ALL was granted on March 29, 2018.

The FDA last month approved two new gene therapies for Duchenne Muscular Dystrophy (DMD) and hemophilia A, and major PBMs tell AIS Health, a division of MMIT, that they have not yet decided how to cover the new treatments. If current trends are any indication, health plans will impose strict utilization management requirements and attempt to negotiate outcomes-based reimbursement pacts with the treatments’ manufacturers.

The FDA on June 22 granted accelerated approval to Sarepta Therapeutics, Inc.’s Elevidys (delandistrogene moxeparvovec-rokl), the DMD treatment. It will have a list price of $3.2 million. BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec), for hemophilia A, gained full FDA approval on June 29, and will have a list price of $2.9 million. Both therapies are administered as a one-time dose. Roctavian also typically requires an indefinite period of simultaneous treatment with corticosteroids to reduce patients’ immune-system response to the gene therapy.