New FDA Approvals: FDA Expands Patient Population of Takeda’s HyQvia

April 11: The FDA expanded the patient population of Takeda’s HyQvia (immune globulin infusion 10% [human] with recombinant human hyaluronidase) to include the treatment of primary immunodeficiency in people between the ages of 2 to 16 years. The agency first approved the drug on Sept. 12, 2014. Subcutaneous dosing can be administered at home or in an infusion center every three or four weeks after a ramping-up period. The volume administered is based on weight and trough level. Drugs.com lists the price of 160 u/mL for 105 milliliters as more than $2,464.

April 14: The FDA expanded the label of Novartis Pharmaceutical Corp. unit Sandoz Inc.’s Hyrimoz (adalimumab-adaz) for the treatment of moderate-to-severe hidradenitis suppurativa in adults. The agency initially approved the biosimilar of AbbVie Inc.’s Humira (adalimumab) on Oct. 30, 2018. Dosing starts with 160 mg via subcutaneous injection on day one or split over two consecutive days, then 80 mg on day 15, and then on day 29 and subsequent doses, 40 mg every week or 80 mg every other week. Drugs.com lists the price of two 40 mg/0.4 mL kits and two 40 mg/0.8 mL kits of Humira, each with two devices, as more than $7,299. Sandoz has said it will launch Hyrimoz on July 1 per the settlement of patent litigation with AbbVie.


Brukinsa Gains Another Approval in Non-Hodgkin Lymphoma

The FDA recently expanded the use of BeiGene, Ltd.’s Brukinsa (zanubrutinib) to include its use in the treatment of a hematologic cancer. The agent is already approved for three other rare types of non-Hodgkin lymphoma. Respondents to a Zitter Insights survey said that while its availability will result in a lower level of unmet need in the treatment of chronic lymphocytic leukemia (CLL), there is still moderate or high unmet need for the condition.

On Jan. 1, the FDA expanded the label of Brukinsa to include the treatment of adults with CLL or small lymphocytic lymphoma (SLL). CLL and SLL are the same disease, a type of non-Hodgkin lymphoma, except CLL cancer cells are mostly in the blood and bone marrow, while in SLL, the cells are mainly in the lymph nodes. CLL is the most common adult leukemia.


News Briefs: Celltrion Launches Vegzelma, Fourth Avastin Biosimilar in U.S.

Celltrion Healthcare Co. launched Vegzelma (bevacizumab-adcd), the company said on April 17. The FDA approved the injectable last September for multiple types of cancer, including colorectal and non-small cell lung cancer. It is the fourth FDA-approved biosimilar of Avastin (bevacizumab) from Genentech USA, Inc., a member of the Roche Group, to launch in the U.S. While the company has partnered with other companies in marketing biosimilars in the U.S., its Celltrion USA unit is taking full responsibility for this launch.

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 11-1 that AstraZeneca and Merck & Co., Inc.’s Lynparza (olaparib) in combination with abiraterone and prednisone or prednisolone for the treatment of metastatic castration-resistant prostate cancer (mCRPC) should be limited to people whose tumors have a BRCA mutation. The FDA accepted a supplemental New Drug Application for a broader approval in mCRPC for the poly (ADP-ribose) polymerase (PARP) inhibitor last August. The class of drugs — which also includes GDK’s Zejula (niraparib) and Rubraca (rucaparib), whose rights Clovis Oncology, Inc. recently sold to pharma& Schweiz GmbH a few months after it filed for Chapter 11 bankruptcy — have been under some scrutiny after they withdrew some of their indications.


CGT Manufacturers Face Variety of Commercialization Challenges

Sessions during the cell and gene therapy (CGT) track at the Reuters Pharma USA conference, held March 28 and 29 in Philadelphia, echoed much of the same advice for pharma companies commercializing one of these products. Starting the process early and collaborating with and forming partnerships with a variety of stakeholders are critical steps to success, multiple speakers emphasized.

Companies looking to bring a cell and gene therapy (CGT) to market should start preparing early on issues such as manufacturing and logistical matters, market access and patient access, advised Warner Biddle, senior vice president and global head of commercial for Kite Pharma, a Gilead Sciences, Inc. company. Kite has two chimeric antigen receptor T-cell therapies on the U.S. market: Yescarta (axicabtagene ciloleucel) — the second CAR-T to secure FDA approval — and Tecartus (brexucabtagene autoleucel).


New First-in-Class Therapy Should Help Ease Disease Burden for People With Follicular Lymphoma

The FDA recently approved a new first-in-class agent for follicular lymphoma, an important development for people with the condition, according to one oncologist. Payers and oncologists both agreed that the drug’s approval should help ease the disease burden somewhat for people suffering from the condition, according to a Zitter Insights survey.

On Dec. 22, the FDA gave accelerated approval to Roche Group member Genentech USA, Inc.’s Lunsumio (mosunetuzumab-axgb) for the treatment of adults with relapsed or refractory follicular lymphoma after at least two lines of systemic therapy. The drug is a first-in-class CD20xCD3 T-cell engaging bispecific antibody. Dosing for the agent — which can be done in an outpatient setting — is 1 mg via intravenous infusion on day one of cycle one, 2 mg on day eight of cycle one and 60 mg on day 15 of cycle one, each over a minimum of four hours. On day one of cycle two, dosing is 60 mg, and then in cycle three and following cycles, dosing is 30 mg on day one; administration can be reduced to two hours if cycle one infusions were tolerated. Dosing for eight cycles is recommended. If people have a partial response or stable disease after eight cycles, an additional nine cycles may be administered. The drug’s price for eight cycles is about $180,000.


People Are Seeing Benefits From Biomarker Testing, but Barriers to Coverage Remain

Biomarker testing is an important tool in cancer care, but a recent survey found payer coverage issues are creating access barriers. According to CancerCare, researchers found that biomarkers helped providers offer personalized care for various cancers for nearly all — 93% — respondents. Twenty percent of surveyed patients were able to forgo unneeded chemotherapy and/or radiation, while 10% found that they were eligible for a clinical trial.

However, the survey also found that 29% of people who had biomarker testing had insurance that did not cover it, prompting them to undergo appeals, obtain financial assistance or pay out of pocket for the service. In addition, 25% of patients said that their insurer required prior authorization (PA) for the process, a tactic that can delay access to treatment.


New FDA Approvals: FDA Approves Acadia’s Daybue for Rett Syndrome

March 10: The FDA approved Acadia Pharmaceuticals Inc.’s Daybue (trofinetide) for the treatment of Rett syndrome in people at least 2 years old. The drug is the only FDA-approved treatment for the complex, rare neurodevelopmental disorder, which is estimated to affect 6,000 to 9,000 people in the U.S. The agency gave the drug fast track status and orphan drug designation; it also gave the company a rare pediatric disease priority review voucher. Dosing of the oral solution is weight based and can be given orally or via gastrostomy tube. The company has not disclosed the agent’s price, but analyst estimates are between $400,000 and $600,000 per year. Acadia said it expects the agent to be available by the end of April.

March 13: The FDA expanded the patient population of Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) to include the treatment of cholestatic pruritus in people with Alagille syndrome who are at least 3 months old. The agency initially approved the oral solution on Sept. 29, 2021. The starting dose is 190 mcg/kg once daily and then increased to 380 mcg/kg after one week. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters as more than $53,712.


Bladder Cancer Gene Therapy Adstiladrin Offers New Option in Slim Category

In late 2022, the FDA approved the first gene therapy for bladder cancer, Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg). Almost three-quarters of oncologists surveyed by Zitter Insights expressed at least moderate interest in the agent, and payers said they expect to manage the drug to label. While the drug gives another treatment alternative to a much-needed area, it likely will continue to put financial pressure on payers, industry experts tell AIS Health, a division of MMIT.

On Dec. 16, the FDA approved Adstiladrin for the treatment of adults with high-risk, Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. The agency gave the novel adenovirus vector-based gene therapy priority review, breakthrough therapy and fast track designations. Dosing is once every three months into the bladder via a urinary catheter. The company said it expects the therapy to be available in the second half of 2023.


Prime, Magellan Studies Reveal Ways to Squeeze More Value From Specialty Drugs

Prime Therapeutics LLC and Magellan Rx Management, a Prime company as of the end of last year, recently presented findings from a series of studies that used integrated medical and pharmacy claims to assess real-world drug use. Their findings show that different strategies can help identify potential member issues that could impact payer costs.

The studies were presented at the Academy of Managed Care Pharmacy (AMCP) Annual Meeting, held in San Antonio, Texas, March 21 to 24.


News Briefs: J&J, AbbVie Units Will Withdraw Two Imbruvica Indications

The Janssen Pharmaceutical Companies of Johnson & Johnson and AbbVie Inc. unit Pharmacyclics LLC are voluntarily withdrawing two indications for Imbruvica (ibrutinib) that had accelerated approval, the companies said on April 6. Following consultation with the FDA, the companies will remove two non-Hodgkin’s lymphoma indications from the drug’s label: the treatment of people with mantle cell lymphoma (MCL) who have received at least one prior therapy, and the treatment of marginal zone lymphoma (MZL) in people who require systemic therapy and have received at least one anti-CD20-based therapy. The decision follows discussion of the results of two Phase III studies — SHINE for MCL and SELENE for MZL — with the FDA, which “advised that the primary outcomes…were considered insufficient to support conversion to full approval.” The Bruton’s tyrosine kinase inhibitor’s other FDA-approved indications, which comprise four disease areas, including three hematologic cancers, are not impacted. The agency first approved the drug on Nov. 13, 2013, when it granted accelerated approval for the MCL indication. It gave accelerated approval for MZL use on Jan. 19, 2017. Since 2020, more than 20 oncology indications and/or drugs approved through the accelerated pathway have been withdrawn.