RADAR on Specialty Pharmacy

FDA Approves Novartis Drug That Will Go Up Against PCSK9s

More than a year after pandemic travel restrictions pushed back the FDA’s approval decision on Novartis Pharmaceuticals Corp.’s inclisiran, the agency finally approved it. The new first-in-class therapy targets so-called bad cholesterol and is set to compete with two other biologics that target the same protein.

On Dec. 22, the FDA approved Leqvio as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with clinical atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH) who require additional lowering of low-density lipoprotein cholesterol (LDL-c).

Biosimilar Approvals Are Expected to Undergo Upward Trend in 2022

The FDA approved only four biosimilars in 2021, down from its high of the 10 it approved in 2019, but slightly more than the three such therapies approved in 2020. The agency also granted interchangeability to the first two biosimilars. This year may up the excitement level, industry experts tell AIS, a division of MMIT, with numerous products expected to be approved, while other noteworthy therapies will lose patent protection and potentially face biosimilar competition.

Dea Belazi, Pharm.D., M.P.H., president and CEO of AscellaHealth: The United States has been slow to approve and adopt biosimilars. Since the initial biosimilar approval in 2015, the FDA has approved 33 biosimilars, but only 18 are currently available on the U.S. market with a projected market value of $13 billion in 2021. Even with this slow uptake in the United States, there is increased utilization and a growing pipeline of products that will drive use and savings. In 2021, many blockbuster drugs were approved in the biosimilar space, along with the two interchangeable biosimilar products, [Boehringer Ingelheim Pharmaceuticals, Inc.’s] Cyltezo and [Viatris Inc. and Biocon Ltd. subsidiary Biocon Biologics Ltd.’s] Semglee.

With New Data, Oncopeptides Rescinds Pepaxto Withdrawal

Three months after Oncopeptides AB said it was voluntarily withdrawing Pepaxto (melphalan flufenamide) from the U.S. market, the company is now taking back that withdrawal and has begun working with the FDA to review new data on the multiple myeloma therapy.

On Jan. 21, the company said that it had contacted the FDA to rescind its Oct. 22 letter requesting voluntary withdrawal of the agent’s New Drug Application (NDA). According to a company press release, “further review and analyses of the heterogenous Overall Survival data from the phase 3 OCEAN study and other relevant trials have led the Company to reconsider its previous voluntary withdrawal request. Oncopeptides has discontinued the marketing of Pepaxto in the US and does not intend to market Pepaxto in the US at this time. The company has initiated a dialogue with the FDA to review the new data.”

Drug Pricing Remains Hot Topic, but Legislation Addressing It Has Stalled

While drug prices continue to be an issue of concern to many Americans, whether it can get any legislative traction issue remains unclear. So what might happen on the issue in 2022?

Dea Belazi, Pharm.D., M.P.H., president and CEO of AscellaHealth: Pharmaceutical drug pricing continues to be a contentious policy issue. Recent data has branded drug list prices growing annually by more than 9% over the last decade, which is much higher than gross domestic product (GDP) growth. However, a RAND study found that prices for unbranded generic drugs...are slightly lower in the United States than in most other nations. Additionally, patients’ out-of-pocket costs for specialty drugs have increased faster than GDP growth over the same period — 2.8% vs. 2.3%. Furthermore, the current White House administration and Congress have declared that reining in Medicare prescription drug costs to help older adults and people with disabilities is a top priority. This will put drug pricing in the crosshairs of elected officials, advocacy groups and patients.…

New FDA Approvals: FDA Grants Additional Indication to Rinvoq

Jan. 14: The FDA expanded the label of AbbVie Inc.’s Rinvoq (upadacitinib) to include the treatment of moderate-to-severe atopic dermatitis in people at least 12 years old whose disease did not respond to previous treatment and is not well controlled with other pills or injections or when those treatments are not recommended. The agency initially approved the Janus kinase (JAK) inhibitor on Aug. 16, 2019. Dosing of the tablet for people weighing at least 40 kg who are at least 12 years old and less than 65 is 15 mg once daily and can be increased to 30 mg once daily. For people at least 65, the recommended dose is 15 mg once daily. Website Drugs.com lists the price of 30 15 mg tablets as more than $5,500.

News Briefs: Gilead Withdraws Two Zydelig Indications

Gilead Sciences, Inc. said on Jan. 14 that it is voluntarily withdrawing two indications for Zydelig (idelalisib): for the treatment of relapsed follicular B-cell non-Hodgkin lymphoma (FL) and small lymphocytic leukemia (SLL), both of which had accelerated approval. “As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” said the company in a press release. Along with the FL and SLL approvals on July 23, 2014, the FDA granted traditional approval to the drug for the treatment of relapsed chronic lymphocytic leukemia. Zydelig will remain on the U.S. market for this indication.

Medicare Plans to Cover Aduhelm but With Certain Restrictions

To say that the FDA’s approval of Biogen and Eisai, Co., Ltd.’s Alzheimer’s disease treatment Aduhelm (aducanumab-avwa) on June 7, 2021, garnered an immense amount of attention would seem to be an understatement. That said, the drug has somehow gathered even more notice over the past few months due to multiple developments, with CMS most recently issuing a proposed National Coverage Determination (NCD) on Aduhelm and other monoclonal antibodies that target beta amyloid plaque that will allow Medicare coverage for the therapies but only under certain circumstances. While commercial payers often follow CMS’s lead, it remains to be seen whether that decision — plus a dramatic price cut on Aduhelm — will prompt payers that have declined to cover the therapy to change course.

There is a 30-day public comment period on the proposed NCD, which was published Jan. 11. A final decision is expected on April 11.

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.

Many Melanoma Therapies Exist, but Treatment Remains Challenging

Since 2011, the FDA has approved multiple therapies for advanced or late-stage melanoma. Recently, the agency granted an additional approval to one of those drugs for the earlier stage melanoma setting, filling an unmet need, industry experts note. However, the condition is complex to treat and may be challenging for health plans to manage.

On Dec. 3, the FDA approved Merck & Co., Inc.’s programmed death receptor-1 (PD-1) inhibitor Keytruda (pembrolizumab) for the adjuvant treatment of people at least 12 years old with stage IIB or IIC melanoma following complete resection. The agency also expanded the indication for the agent’s use as an adjuvant treatment of stage III melanoma following complete resection to include pediatric patients at least 12 years old.

Specialty Pharmacy, Home Infusion Spaces Saw Myriad 2021 Changes

While the COVID-19 pandemic continued to impact the specialty pharmacy and home infusion spaces for the second straight year, other events also played a role. AIS Health spoke to some industry experts about 2021’s impact.

AIS Health: Looking back over the past year, what do you think were the most noteworthy occurrences within the specialty pharmacy industry, and why?

Dea Belazi, Pharm.D., M.P.H., president and CEO of AscellaHealth: Some of the noteworthy trends in SP have included the continued rise of the cost of specialty agents within health care expenditures, the continued emergence of ultra-high-cost specialty agents for rare and orphan diseases and the continued vertical integration of specialty pharmacies into health care organizations.