Drug Approvals

FDA Grants Full Approval to Game-Changing Regimen for Urothelial Cancer

Late last year, the FDA approved a new regimen for urothelial cancer that could transform the treatment of the condition, according to industry experts. However, the cost of the combination — which is estimated to be more than half a million dollars annually — could pose a barrier to its uptake.

On Dec. 15, 2023, the FDA approved Pfizer Inc. and Astellas Pharma Inc.’s Padcev (enfortumab vedotin-ejfv) in combination with Merck & Co., Inc.’s Keytruda (pembrolizumab) for the treatment of people with locally advanced or metastatic urothelial cancer (la/mUC). The agency gave accelerated approval to this combination for people with la/mUC who are not eligible for cisplatin-containing chemotherapy on April 3, 2023. The newest approval converts that accelerated approval to full and expands the labeled indication to include the treatment of people eligible for cisplatin chemotherapy. The application had priority review and breakthrough therapy designation.

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New FDA Approvals: FDA Approves First Generic of Emflaza

Feb. 9: The FDA approved Aurobindo Pharma Limited’s deflazacort for the treatment of Duchenne muscular dystrophy (DMD) in people at least 5 years old. The corticosteroid is the first generic of PTC Therapeutics, Inc.’s Emflaza that the agency has approved. It has approval as a tablet for all of Emflaza’s dosage strengths: 6 mg, 18 mg, 30 mg and 36 mg. Emflaza also is available as an oral suspension. That agent is approved for the treatment of DMD in people at least 2 years old; it has exclusivity for the treatment of people ages 2 to 5 years old until June 2026. The recommended daily dosing is 0.9 mg/kg. Drugs.com lists the price of 30 18 mg tablets of Emflaza as more than $9,706.

Feb. 9: The FDA approved Takeda Pharmaceuticals U.S.A., Inc.’s Eohilia (budesonide) for the treatment of people at least 11 years old with eosinophilic esophagitis. The corticosteroid is the first and only FDA-approved oral treatment for the indication. Recommended dosing is 2 mg twice daily for 12 weeks; the agent is available in 2 mg/10 mL single-dose stick packs. Its monthly wholesale acquisition cost is $1,875.

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New FDA Approvals: FDA Broadens Keytruda Use in Cervical Cancer

Jan. 12: The FDA broadened the label of Merck & Co., Inc.’s Keytruda (pembrolizumab) to include, in combination with chemoradiotherapy, the treatment of people with FIGO (International Federation of Gynecology and Obstetrics) 2014 Stage III-IVA cervical cancer. The agency initially approved the programmed death receptor-1 (PD-1) inhibitor on Sept. 4, 2014. The indication is the drug’s third in cervical cancer and 39th overall. The application was granted priority review. That review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration and the Brazilian Health Regulatory Agency, and it used the Assessment Aid. Dosing is 200 mg every three weeks or 400 mg every six weeks via a 30-minute intravenous infusion. The agent’s price for every-three-weeks dosing is $11,115; for every-six-weeks dosing, it’s $22,230.

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Number of FDA Approvals Increased in 2023, With Notable Agents Including Cell and Gene Therapies

In 2023, the FDA continued to rebound from a drop in approvals, marking the highest number in years. The agency’s Center for Drug Evaluation and Research (CDER) approved 55 novel drugs last year, and its Center for Biologics Evaluation and Research (CBER) approved 17 agents. That’s up from 37 CDER-approved therapies in 2022 and 51 in 2011. In 2022, CBER OK’d 13 agents, up from 10 in 2021. Specialty agents, such as cell and gene therapies, continued to make up a large portion of those new approvals, while the FDA approved several biosimilars, including a handful that were the first versions of their reference drugs. AIS Health, a division of MMIT, spoke with industry experts about what they view as the most notable FDA approvals of 2023.

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Study: Medicare Advantage Members Are More Likely to Use Biosimilars

Biosimilar uptake is higher for Medicare Advantage beneficiaries than their traditional Medicare (TM) counterparts, with MA members up to 2.3 times more likely to be prescribed a biosimilar, according to new research published in JAMA Health Forum. The FDA has approved 45 biosimilars so far, with more approvals expected this year.

Researchers from CMS, the FDA and policy research firm Acumen LLC studied claims and encounter data to calculate market share for 20 biosimilars across seven product categories. Biosimilar uptake was higher for MA members in every category but bevacizumab, the generic name for Genentech’s targeted cancer therapy Avastin. Avastin currently has four biosimilars available, with a fifth approved last month.

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What’s Next For ICER: An Interview With New President Sarah Emond

The Institute for Clinical and Economic Review’s (ICER’s) new president and CEO Sarah Emond took over the role from the organization’s longtime leader and founder Steve Pearson on Jan. 1, and in an interview at the J.P. Morgan Healthcare Conference in San Francisco, she discussed some of the pressing drug pricing issues on ICER’s radar for the year ahead.

Emond has had a lengthy career at ICER, having worked for the organization for 14 years — most recently as vice president and chief operating officer, helping to lead strategic operations. She said the leadership change was part of a long and planned transition and Pearson continues to work as an advisor.

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Study: Medicare Advantage Members Are More Likely to Use Biosimilars

Biosimilar uptake is higher for Medicare Advantage beneficiaries than their traditional Medicare (TM) counterparts, with MA members up to 2.3 times more likely to be prescribed a biosimilar, according to new research published in JAMA Health Forum. The FDA has approved 45 biosimilars so far, with more approvals expected this year.

Researchers from CMS, the FDA and policy research firm Acumen LLC studied claims and encounter data to calculate market share for 20 biosimilars across seven product categories. Biosimilar uptake was higher for MA members in every category but bevacizumab, the generic name for Genentech’s targeted cancer therapy Avastin. Avastin currently has four biosimilars available, with a fifth approved last month.

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Obesity Meds, Gene Therapies, NASH Drug Make Payers’ Cost Concern List

For years, payers have been concerned about the rising prices of prescription medications and how to cover newly approved drugs. Pharmaceutical experts tell AIS Health, a division of MMIT, that PBMs and plans will continue to be challenged in 2024 with similar issues, particularly when it comes to gene therapies, obesity medications and other expensive products.

Andy Szczotka, Pharm.D., chief pharmacy officer at AscellaHealth, notes that more than half of pharmacy benefit spending is on specialty medications even though only a small percentage of members use those drugs.

He says dealing with high-cost specialty products “is a focus for most payers” and adds there is a “large target on specialty drugs.”

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New FDA Approvals: FDA Expands Bivigam Patient Population

Dec. 8: The FDA expanded the patient population of ADMA Biologics, Inc.’s Bivigam (immune globulin intravenous, human) to include the treatment of people at least 2 years old with primary humoral immunodeficiency. The agency initially approved the therapy on Dec. 21, 2012. The initial intravenous infusion rate is 0.5 mg/kg/minute for the first 10 minutes for a dose of 300-800 mg/kg every three to four weeks. The maintenance infusion rate may be increased every 20 minutes by 0.8 mg/kg/minute up to 6 mg/kg/minute. Drugs.com lists the price of a 50 mL intravenous solution as more than $756.

Dec. 13: The FDA approved US WorldMeds’ Iwilfin (eflornithine) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-disialoganglioside (anti-GD2) immunotherapy. Between 700 and 800 cases of neuroblastoma are diagnosed annually in the U.S., with 90% of those before age 5. About half of those cases in children are HRNB. The agency used the Real-Time Oncology Review pilot program and the Assessment Aid. It also granted the application priority review and breakthrough drug and orphan drug designations. The recommended dosing of the tablet is based on body surface area.

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2023 Notable FDA Approvals Include Several Cell and Gene Therapies, First-Time Biosimilars

This past year, the FDA continued to rebound from a drop in approvals, marking the highest number in years. The agency’s Center for Drug Evaluation and Research (CDER) approved 55 novel drugs last year, and its Center for Biologics Evaluation and Research (CBER) approved 17 agents. That’s up from 37 CDER-approved therapies in 2022 and 51 in 2011. In 2022, CBER OK’d 13 agents, up from 10 in 2021. Specialty agents, such as cell and gene therapies, continued to make up a large portion of those new approvals, while the FDA approved several biosimilars, including a handful that were the first versions of their reference drugs. AIS Health, a division of MMIT, spoke with industry experts about what they view as the most notable FDA approvals of 2023.

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