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Articles

Check out these important FDA updates from the month of July 2024.

FDA Roundup: Agency Highlights from July 2024

Researchers aimed to address the associations between prenatal cannabis use and maternal health outcomes over a 9-year period.

Prenatal Cannabis Use and Maternal Health Outcomes

Slideshows

The American College of Allergy, Asthma, and Immunology (ACAAI) recently released its 5 tips to help children and their parents stay educated on asthma peak month.

Slideshow: Managing The Peak of Asthma Season

Galderma said the FDA has accepted the company’s Biologics License Application for the therapy based on data from the trials.

Nemolizumab for Atopic Dermatitis Shows Positive Results in Phase 3 Trials

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Clinical

Practical Approaches in the Management of Primary Immunodeficiency Disorders: Understanding the Role of Intravenous and Subcutaneous Immunoglobulin 

1.5 Credits / Cardiology, Neurology, Rare Diseases

Exploring Transthyretin-Mediated Amyloidosis and Its Phenotypes: A Comprehensive Approach to Patient Treatment

Fidanacogene elaparvovec-dzkt (Beqvez) is a 1 time treatment that allows patients with hemophilia B to produce factor IX themselves.

FDA Approves Pfizer’s Gene Therapy for Hemophilia B

The company’s investigational ABCWY vaccine candidate will be reviewed by the FDA by February 14, 2025.

FDA Accepts GSK's BLA for 5-in-1 Meningococcal Vaccine

Data will be discussed with regulatory authorities “in the coming months.” 

Pfizer Hemophilia A Candidate Shows Positive Topline Results

The approval of eculizumab-aagh offers more treatment options for patients with rare diseases PNH and aHUS.

FDA Approval Marks Epysqli as Second Soliris Biosimilar

The drug was FDA approved as antihemophilic factor (recombinant), Fc-VWF-XTEN Fusion Protein-ehtl (Altuviiio) in February 2023.

Efanesoctocog alfa Prophylaxis Offers “Efficacious Prevention” to Children With Severe Hemophilia A

Eculizamab-aeeb is the 53rd FDA-approved biosimilar in the US.

Amgen Gains FDA Approval for First Interchangeable Biosimilar to Eculizumab

WHIM syndrome is characterized by low levels of neutrophils and lymphocytes in the blood due to dysfunction in the CXCR4 receptor, which results in serious and frequent infections.

FDA Approves Mavorixafor for Treatment of WHIM Syndrome

Paying for these new therapies, that may be curative for patients, remains a challenge.

The Pipeline Is Full of Expensive, Innovative Therapies

The pipeline is full of expensive, but innovative therapies, and finding room in the budget to cover them continues to be a challenge, explained Jon Mahrt, MBA, president of the pharmacy benefit manager, and chief operating officer of OptumRX.

The trend over the last 7 years has been encouraging for orphan drug approvals, and another 20 are expected this year.

“…if you’re a rare disease patient, these are incredibly exciting times,” Mahrt said.

However, the high cost of these treatments remains a challenge, with some drugs costing over $2 million. These prices don’t are only the wholesale acquisition cost and don’t take into account other costs of treatment, such as supply chain logistics and procedural challenges for physicians and pharmacists, which can drive up the cost even more.

Since these drugs are potentially curative, the prices of other drugs in the portfolio need to be reduced to create room for the curative therapies to come to market, he said.

“If you look at the cost to manufacture these drugs, they're a fraction” of the wholesale acquisition cost, Mahrt said. He added that the Institute for Clinical and Economic Review analyzed unsupported price increases of drugs in 2022, and 8 of 10 drugs evaluated had unsupported pricing, meaning there was no new evidence and yet the price increased above inflation.

The report had found these increases accounted for $1.27 billion in additional costs over 1 year.

“So, there's plenty of money there when these drugs come to market at market prices to fund future innovation,” Mahrt said. “I think it’s…focusing on the right drug for the right patient, and that will ensure we get forward-looking innovation in the market.”

 at AXS24, the Asembia Specialty Pharmacy Summit, held April 28 to May 2 in Las Vegas, Nevada. 

Click here for more of our coverage from AXS24

1. Rind DM, Agboola F, Nikitin D, et al. Unsupported price increase report: unsupported price increases occurring in 2022. December 11, 2023. Accessed April 30, 2024. 

https://icer.org/wp-content/uploads/2023/12/UPI_2023_Report_121123.pdf

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