Radar on Specialty Pharmacy

In December, the FDA approved the two newest cell and gene therapies, which were the first such agents approved for the treatment of sickle cell disease. As more of these products launch onto the U.S. market — the agency previously predicted that it would be approving 10 to 20 of the treatments by 2025 — payers are taking a variety of approaches to managing the therapies. Cost remains the main obstacle to their use, but their long-term durability also remains a question to some extent.

Dec. 8 saw the newest approvals, both for the treatment of sickle cell disease in people at least 12 years old: bluebird bio, Inc’s Lyfgenia (lovotibeglogene autotemcel; lovo-cel) and Vertex Pharmaceuticals Inc. and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel; exa-cel). The latter agent is the first CRISPR/Cas9 genome-edited cell therapy that the FDA has approved. The FDA gave both applications priority review, orphan drug, fast track and regenerative medicine advanced therapy designations. It also gave Lyfgenia rare pediatric disease designation.

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.

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.

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.