Oncology

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.

0 Comments
© 2024 MMIT

AI Use in Pharma Shows Promise, Prompts Caution

It’s hard to underestimate the reach of artificial intelligence (AI) across the health care and pharmaceutical industries. While the ultimate impact of the technology on payers and providers may be debatable, pharma companies have made broader inroads into exploring ways to enhance their efforts, including in drug discovery. Still, caution in some areas is warranted, according to some studies.

For the Managed Care Biologics & Injectables Index: Q3 2023, from Aug. 13, 2023, to Sept. 29, 2023, Zitter Insights polled 35 commercial payers covering 117.7 million lives, 103 physicians and 83 practice managers about their familiarity with Open AI’s ChatGPT and AI tools in general.

0 Comments
© 2024 MMIT

Industry Veteran Suggests Broader Genomics Use for Proactive Approach to Health Care

Tremendous strides have been made in personalized medicine over the past several years — not only in drugs but in the various biomarkers used to identify the right drug for the right patient at the right time. While many people may think of this approach as one mainly solely for cancer, the potential for personalized medicine to have a broader impact on health outcomes when it is used proactively has not fully been realized.

In September, InformedDNA — which provides genomic solutions to an array of health care stakeholders to improve outcomes — acquired gWell Health, a digital health, genomics and wellness company. As part of the deal, gWell founder and CEO Surya Singh, M.D., transitioned to CEO of InformedDNA. Singh has worked in various health care organizations in more than 20 years, including serving as corporate vice president and chief medical officer at CVS Health Specialty.

0 Comments
© 2024 MMIT

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.

0 Comments
© 2024 MMIT

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.

0 Comments
© 2024 MMIT

News Briefs: Elevance Health Will Acquire Paragon Healthcare

Elevance Health, Inc. has agreed to acquire infusion services provider Paragon Healthcare, Inc., the health insurer said Jan. 4. The companies did not disclose financial details of the deal, which is expected to close in the first half of 2024. Paragon Healthcare serves more than 35,000 people at more than 40 ambulatory infusion centers in eight states, as well as in the home setting, and it treats more than 300 conditions. Once the deal is finalized, Paragon Healthcare will operate as part of CarelonRx, which is the pharmacy segment within Elevance Health’s Carelon health services division. The deal follows Elevance Health’s acquisition of BioPlus, a specialty pharmacy subsidiary of CarepathRx, a portfolio company of Nautic Partners, which it closed in February 2023.

Hizentra (immune globulin subcutaneous [human] 20% liquid) is now available in a 10 g prefilled syringe, manufacturer CSL Behring disclosed Jan. 3. The agent is the first and only subcutaneous immune globulin treatment approved for the maintenance of chronic inflammatory demyelinating polyneuropathy in the U.S. It also is approved for primary immunodeficiency in people at least 2 years old. In addition to the new size, the drug also is available in 1 g, 2 g and 4 g prefilled syringes.

0 Comments
© 2024 MMIT

New FDA Approvals: FDA Grants Additional Indication to Orencia

Oct. 30: The FDA gave an additional approval to Bristol Myers Squibb’s Orencia (abatacept) for the subcutaneous treatment of people at least 2 years old with active psoriatic arthritis. The agency first approved the selective T cell costimulation modulator on Dec. 23, 2005. Dosing for the newest use is 50 mg once weekly for people with a body weight of 10 kg to less than 25 kg, 87.5 mg for those with a body weight of 25 kg to less than 50 kg and 125 mg for those with a body weight of at least 50 kg. The agent also is approved for intravenous dosing of certain indications. GoodRx lists one carton of four 125 mg/mL single-dose syringes as more than $5,520.

Oct. 31: The FDA expanded the approval of Fresenius Kabi’s Idacio (adalimumab-aacf) for the treatment of adults with non-infectious intermediate and posterior uveitis and panuveitis. The agency first approved the biosimilar of AbbVie Inc.’s tumor necrosis factor (TNF) blocker Humira (adalimumab) on Dec. 13, 2022. Dosing starts with 80 mg via subcutaneous injection, followed by 40 mg every other week starting one week after the initial dose. The price of two single-dose prefilled pens or two single-dose prefilled glass syringes is $6,576.

0 Comments
© 2024 MMIT

News Briefs: HHS Unveils Draft Guidance on Using March-In Rights

On Dec. 8, the U.S. Department of Commerce’s National Institute of Standards and Technology and HHS unveiled draft guidance (88 Fed. Reg. 85593) on framework for exercising march-in rights on taxpayer-funded drugs and other products. The framework “specifies that price can be a factor in considering whether a drug is accessible to the public,” the administration said. That was one of other “new actions to promote competition in health care and support lowering prescription drug costs for American families.” Comments must be received by 5 p.m. Eastern on Feb. 6.

0 Comments
© 2024 MMIT

Another PI3K Inhibitor Will Be Withdrawn From U.S. Market

Bayer recently revealed that it will work with the FDA to voluntarily withdraw the New Drug Application (NDA) for its cancer drug Aliqopa (copanlisib). The therapy is the latest phosphoinositide 3-kinase — also called phosphatidylinositol 3-kinase — (PI3K) inhibitor/indication with accelerated approval to treat a hematologic malignancy to be pulled from the U.S. market, potentially spurring payers to take a closer look at these agents.

The agency gave Aliqopa accelerated approval on Sept. 14, 2017, for the treatment of adults with relapsed follicular lymphoma (FL) who have received at least two systemic therapies. Approval was based on the CHRONOS-1 Phase II clinical trial. In the confirmatory study, CHRONOS-4, adding Aliqopa to standard immunochemotherapy regimens did not meet the primary endpoint of progression-free survival (PFS) vs. the standard immunochemotherapy control arm. Bayer says it will publish the trial results “in a timely manner.”

0 Comments
© 2024 MMIT

Scrutiny of Accelerated Approval Continues as FDA Clamps Down

Scrutiny of the FDA’s accelerated approval process has shown no signs of slowing, with an FDA committee holding a recent meeting on the program’s use for oncology indications, as well as on two cancer drugs lacking confirmatory trial data long after their accelerated approvals. And an FDA leader recently asserted his stance on granting the designation only when companies already have started postmarketing trials. Still, these developments should not dampen interest by either manufacturers or payers in drugs hitting the market via the accelerated pathway, says one industry expert.

The FDA established the accelerated approval pathway in 1992 to bring HIV/AIDS medicines onto the market sooner during the ongoing epidemic. Between that time and Dec. 31, 2020, the agency has granted more than 253 accelerated approvals, according to a 2021 report from the Institute for Clinical and Economic Review (ICER) examining the designation. Of those, 125 — 49.4% — later received full approval, with a median time of 3.2 years from accelerated approval to full. Sixteen — 6.3% — of the indications have been withdrawn, and the remaining 112 drugs have been on the market for a median of 1.9 years.

0 Comments
© 2024 MMIT