FDA

Amneal Pharmaceuticals, Inc. launched Fylnetra (pegfilgrastim-pbbk), the company said May 16. The granulocyte colony-stimulating factor is the sixth biosimilar of Amgen Inc.’s Neulasta (pegfilgrastim) to launch in the U.S. The FDA approved the drug on May 26, 2022, to decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. Its J-code is Q5130. It is the third biosimilar that Amneal has launched in the U.S.

Amgen settled a patent infringement lawsuit (No. 22-cv-1549) with Janssen Biotech Inc., a division of Johnson & Johnson, over a proposed biosimilar of Stelara (ustekinumab), Amgen revealed May 23. Stelara, a human interleukin-12 and -23 antagonist, is indicated for adults with moderate-to-severe plaque psoriasis, moderately to severely active Crohn’s disease and moderately to severely active ulcerative colitis, as well as people at least 6 years old with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy and people at least 6 with active psoriatic arthritis. The FDA first approved it on Sept. 25, 2009. Amgen said the settlement will allow its biosimilar to launch “no later than” Jan. 1, 2025.

May 9: The FDA approved Chiesi Group unit Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc.’s Elfabrio (pegunigalsidase alfa-iwxj) for the treatment of adults with Fabry disease. A PEGylated enzyme replacement therapy, the agent is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life. Recommended dosing is 1 mg/kg via intravenous infusion every two weeks.

May 17: The FDA approved a 40 mg/0.8 mL single-dose autoinjector for Boehringer Ingelheim’s Cyltezo (adalimumab-adbm). The agency initially approved the biosimilar of AbbVie Inc.’s Humira (adalimumab) on Aug. 25, 2017, as a single-use prefilled syringe. The drug is approved for multiple chronic inflammatory diseases. The new pen will be available in two-, four- and six-pack options when the tumor necrosis factor (TNF) inhibitor launches on July 1.

Eisai Co., Ltd. and Biogen Inc.’s Leqembi (lecanemab) could raise Medicare Part B spending by $8.9 billion or more annually if it receives full approval from the FDA KFF found. And a report by researchers from the RAND Corp. found that Leqembi could increase spending by $2 billion to $5 billion. KFF’s projection was based on an annual list price of $26,500 and uptake among 5% of Alzheimer’s patients. The RAND report included estimates of ancillary costs, such as MRI scans. The FDA granted accelerated approval to the anti-amyloid monoclonal antibody treatment in January, and its decision on full approval is expected soon.

CVS Health Corp is shutting down its clinical trials division, which launched in 2021, and will conclude operations in 2024. The division has worked with more than 30 drugmakers on 50 Phase I, II and IV studies involving 33,000 participants, according to Modern Healthcare.

GlaxoSmithKline Pharmaceuticals Ltd. won the first US approval for a respiratory syncytial virus vaccine on May 3 in older adults but looks like it may not be able to claim the same crown in the other key population impacted by the virus — infants and young children.

The Food and Drug Administration approval letter for GSK’s Arexvy says the agency is waiving the pediatric study requirement for children ages 0 to 2 “because there is evidence strongly suggesting that the biological product would be unsafe in this pediatric group.”

The label for Arexvy, which is indicated for adults 60 and older, says “evidence from an animal model strongly suggests that Arexvy would be unsafe in individuals younger than 2 years of age because of increased risk of enhanced respiratory disease.”

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.

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.

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.

While the cell and gene therapy (CGT) industry has shown remarkable progress, it still has some barriers to overcome. The FDA is taking numerous steps to help bring these products onto the market, asserted Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER), the unit that oversees biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies, at a recent public appearance.

“I think the bottom line here for us is that we are very committed to helping move this field forward for populations of all sizes,” particularly for small populations, which is “one of the areas that is our highest medical need right now,” said Marks during a session in the CGT track at the Reuters Pharma USA conference, held March 28 and 29 in Philadelphia.

Oklahoma lawmakers accused CVS Health Corp.’s Caremark PBM of “intentionally lying” in its denials of 90-day prescription fills for some patients, according to The Oklahoman. The Oklahoma City newspaper reported that Caremark had routinely denied Oklahoma residents 90-day prescription fills and cited a state law as the reason, claiming that such fills were illegal under the statute. State Rep. Jon Echols, a Republican, said that such a justification was a lie: “Anyone that says that is not misinformed. They don’t misunderstand. They are intentionally lying to you. And we’re not going to stand for it.” Oklahoma’s insurance commissioner, Republican Glen Mulready, earlier this month filed an administrative action against Caremark that, per a press release from Mulready’s office, “targets the practice of ‘steering patients’ to CVS pharmacies and prescription mail-order services and seeks to censure, suspend, place on probation or revoke the Pharmacy Benefit Manager (PBM) license of CVS/Caremark. In addition, the… Department will seek restitution and/or levy fines for each alleged violation.”

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).