Cell and Gene Therapies

Carvykti Approval Brings Second CAR-T to Multiple Myeloma Treatment

With its Feb. 28 approval, the Janssen Pharmaceutical Companies of Johnson & Johnson and Legend Biotech USA, Inc.’s Carvykti (ciltacabtagene autoleucel; cilta-cel) becomes the second chimeric antigen receptor T-cell (CAR-T) therapy to treat multiple myeloma. Payers report being less likely to prefer it over some or all other multiple myeloma treatments with a similar indication, but oncologists are showing more enthusiasm for prescribing the new agent, according to Zitter Insights.

The FDA approved Carvykti for the treatment of adults with relapsed or refractory multiple myeloma after at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody. The product is a B-cell maturation antigen (BCMA)-directed CAR-T agent. The one-time treatment will have a phased launch and will be available through a limited network of certified treatment centers. The FDA gave the drug breakthrough therapy and orphan drug designations. The therapy’s wholesale acquisition cost is $465,000.

New FDA Approvals: The FDA Granted an Additional Indication to Lynparza

March 11: The FDA granted an additional indication to AstraZeneca and Merck & Co., Inc.’s Lynparza (olaparib) for the treatment of people with germline BRCA-mutated human epidermal growth factor receptor 2 (HER2)-negative high-risk early breast cancer who have been treated with chemotherapy before or after surgery. The agency first approved the tablet on Dec. 19, 2014. Dosing is 300 mg twice daily. Website Drugs.com lists the price of 60 150 mg tablets as more than $7,778.

March 11: The FDA approved another use for Myriad Genetics, Inc.’s BRACAnalysis CDx test as a companion diagnostic to identify people with germline BRCA-mutated HER2-negative, high-risk early-stage breast cancer who may benefit from Lynparza (see above brief). The test detects and interprets germline BRCA1 and BRCA2 variants. The agency initially approved the test on Dec. 19, 2014.

BCBS of Michigan Launches Precision Medicine Pilot Program

Blue Cross Blue Shield of Michigan last month launched a precision medicine pilot program for 500 of its Medicare Advantage HMO members who also have pharmacy benefits. Experts say the move is becoming more common among payers as they look to reduce their medication spending and improve clinical outcomes in high-risk groups.

The program, Blue Cross Personalized Medicine, is using pharmacogenomics (PGx), or genetic testing, to help identify how people respond to certain medications and offer specific treatment recommendations based on their genetic makeup. Its aim is to help BCBS of Michigan manage high drug costs and give its members tailored, cost-effective and clinically relevant care while also decreasing adverse drug reactions.

Within Innovative Oncology Space, Companies Need to Address Oncologists’ Needs

The oncology space is undergoing a tremendous amount of innovation, as novel new products and practices become available. But those treatments can do only so much good if oncologists aren’t using them. Biopharma companies have an opportunity to differentiate themselves from their competitors by addressing oncologists’ specific needs, industry experts tell AIS Health, a division of MMIT.

When it comes to drug information, oncologists not only want to understand a product’s efficacy, “but also how to efficiently and effectively diagnose the patient and get that patient to the right targeted drug or combination using the patient’s genetics and the genetics of the tumor,” such as BRCA1 mutation-positive in breast cancer, explains Kristen Pothier, principal at KPMG U.S. Healthcare and life sciences deal advisory and strategy leader.

2022 Outlook: Notable FDA Approvals, Payer Strategies Are Specialty Trends

The specialty pharmacy industry will continue its growth in 2022, experts tell AIS Health, a division of MMIT. Multiple agents for rare diseases are poised for FDA approval, and the market may see additional cell and gene therapies. In addition, payers will carry on their efforts to keep prices for specialty medications affordable, among other trends.

AIS Health: What are some specialty pharmacy issues to keep an eye on in 2022, and why?

Notable 2021 Approvals Included Oncolytics, Interchangeables

Between the FDA’s Center for Drug Evaluation and Research (CDER) and its Center for Biologics Evaluation and Research (CBER), the agency approved 60 new products in 2021, including biologics, cell and gene therapies, vaccines and blood products. As the COVID-19 pandemic continued for the second year, much of the pharma industry’s focus, understandably, was on vaccines and therapeutics for the coronavirus. As with prior years, oncology dominated the specialty drug space, but the FDA also approved novel treatments in other areas as manufacturers continued to pursue innovative new products. Moreover, the U.S. saw the first two interchangeable biosimilars gain approval. AIS Health, a division of MMIT, spoke with various industry experts about what they considered the most notable FDA approvals in 2021.

Looking Forward to 2022, COVID-19 Will Continue to Impact Pharma in Myriad Ways

As 2021 began, the pharmaceutical topic arguably drawing the most attention was COVID-19 and the rollout of vaccines against it. Moving into 2022, the COVID pandemic unfortunately continues to dominate headlines, as the U.S. marked 1 million new cases of COVID on Jan. 3, a single-day record for any country. However, it’s not all bad news, as the pandemic is continuing its impact across various services. For example, drugmakers are applying knowledge gained in their innovative efforts to develop vaccines and treatments to other areas of drug development, and a shift to virtual operations and health care is expected to continue.

AIS Health spoke with a variety of industry experts about their 2022 projections for pharma.

New Tecartus FDA Approval for Adults With ALL Offers Welcome Therapeutic Option

On Oct. 1, the FDA gave another indication to Gilead Sciences, Inc. division Kite Pharma, Inc.’s Tecartus (brexucabtagene autoleucel) for the treatment of people 18 years and older with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). According to a Zitter Insights survey, oncologists said they are likely to prescribe the drug over certain therapies for that indication. Industry experts tell AIS Health, a division of MMIT, that the agent may be a game changer for certain patients, as it fills a treatment gap.

News Briefs: Oncopeptides AB is withdrawing Pepaxto from the U.S. market | Nov. 16, 2021

Oncopeptides AB is voluntarily withdrawing Pepaxto (melphalan flufenamide) from the U.S. market, the company disclosed on Oct. 22. The FDA gave the therapy accelerated approval on Feb. 26, 2021, in combination with dexamethasone for the treatment of adults with relapsed or refractory multiple myeloma who have received at least four lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one CD38-directed monoclonal antibody. The company said that the decision follows the Phase III OCEAN study, which “the FDA does not consider…meets the criteria of a confirmatory study.”