FDA Updates for Week of Aug. 1, 2022

FDA approves Enhertu for HER2 low breast cancer.

After a quick review, the FDA has approved Enhertu (fam-trastuzumab-deruxtecan-nxki), an IV infusion for the treatment of patients with unresectable or metastatic HER2-low breast cancer. This is the first approved therapy targeted to patients with the HER2-low breast cancer subtype, which is a newly defined subset of HER2-negative breast cancer.

The agency had accepted the supplemental biologics license application in July 2022, and the company had indicated that the Prescription Drug User Fee Act date action date was during the fourth quarter of the 2022.

Enhertu is an engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.

FDA approves first interchangeable biosimilar to Lucentis.

The FDA has approved Cimerli (ranibizumab-eqrn) as a biosimilar product interchangeable with Genentech’s Lucentis (ranibizumab). Coherus BioSciences plans to launch the ophthalmologic therapy in early October 2022.

Cimerli will be available in both 0.3 mg and 0.5 mg dosages and will be interchangeable for all of Lucentis’s indications, including neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, diabetic retinopathy, and myopic choroidal neovascularization.

The first biosimilar with interchangeability status compared with its reference product is entitled to one-year of exclusivity of the interchangeability designation.

Coherus didn’t provide a price for Cimerli, but another Lucentis biosimilar, Byooviz, has a list price of $1,130 per single use vial, which is 40% lower than the current list price of Lucentis. Byooviz, developed by Biogen and Samsung Bioepis, launched in June 2022 but it doesn’t have an interchangeable approval.

FDA issues complete response letter for Nuplazid in Alzheimer’s psychosis.

Acadia Pharmaceuticals has received a complete response letter (CRL) from FDA regarding its supplemental new drug application (sNDA) for Nuplazid (pimavanserin) to treat patients with hallucinations and delusions associated with Alzheimer’s disease psychosis. The FDA approved Nuplazid in April 2016 for the treatment of patients with hallucinations and delusions associated with Parkinson’s disease psychosis.

Regulators indicated that one of the studies submitted showed statistical significance for the Alzheimer’s indication, but they agreed with the June 2022 FDA advisory committee conclusion that the evidence from both trials that were submitted doesn’t support approval of Nuplazid in patients with Alzheimer’s.

The FDA and the advisory committee both recommended that Acadia conduct an additional trial in patients with Alzheimer’s psychosis. Acadia officials, however, have said they don't plan to conduct another study.

FDA approves steroid-free Zoryve for plaque psoriasis.

The FDA has approved the new drug application (NDA) for Zoryve (roflumilast) cream for the treatment of plaque psoriasis in patients 12 years of age or older. Zoryve is a once-daily, steroid-free cream and is the first topical phosphodiesterase-4 (PDE4) inhibitor approved for this indication.

Developed by Arcutis Biotherapeutics, Zoryve will be available by mid-August. Arcutis will offer a patient support program to help commercially insured patients get access and help navigate the payer process. The company will also offer the Arcutis Cares patient assistance program – the first of its kind for a topical psoriasis treatment – that will provide Zoryve at no cost for financially eligible patients who are uninsured or underinsured.

FDA approves Stelara in children with psoriatic arthritis.

The FDA has approved Stelara (ustekinumab) to treat children six years of age and older with active psoriatic arthritis (PsA). Stelara is a fully human monoclonal antibody that selectively inhibits both interleukin (IL)-12 and IL-23, two cytokines thought to play an important role in inflammatory response in several autoimmune diseases.

The Janssen Pharmaceutical Companies of Johnson & Johnson submitted a supplemental biologics license application to the FDA in October 2021. The submission is based on data extrapolated from a total of nine studies in adult trials in active psoriatic arthritis and adult and pediatric studies in moderate-to-severe plaque psoriasis.

Stelara is approved in the United States for the treatment of: adults and children 6 years and older with moderate-to-severe plaque psoriasis; adult patients with active psoriatic arthritis; adult patients with moderately to severely active Crohn’s disease or ulcerative colitis.

FDA accepts BLA for biosimilar of Actemra.

The FDA has accepted for Fresenius Kabi biologics license application (BLA) for a biosimilar of Genentech’s Actemra (tocilizumab), a monoclonal antibody that is used to treat patients with moderate-to-severe rheumatoid arthritis. Additionally, the FDA has issued an emergence use authorization for Actemra to treat patients who are hospitalized with COVID-19.

Fresenius Kabi expects to receive FDA approval for its tocilizumab biosimilar in 2023 and is seeking approval for both intravenous and subcutaneous routes of administration. The BLA includes presentations for both subcutaneous (prefilled syringe and autoinjector) and intravenous administrations.

Genentech’s Actemra is under review by the FDA for full approval for treatment of COVID-19 in hospitalized adults who are receiving systemic corticosteroids and require supplemental oxygen. During the COVID-19 pandemic, Genentech struggled with supply shortages, but as of July 2022, the company announced it now has a stable supply.

FDA schedules second advisory committee meeting for AMX0035 in ALS.

The FDA has scheduled a meeting of the Peripheral and Central Nervous System Drugs Advisory Committee on Sept. 7, 2022, to review the new drug application (NDA) for Amylyx Pharmaceuticals’ AMX0035 (sodium phenylbutyrate and taurursodiol). This is the second meeting of the committee to discuss this application and the data relating to AMX0035 to treat patients with amyotrophic lateral sclerosis (ALS).

This meeting will discuss the additional analyses of data from the company’s clinical studies. As a result, FDA extended the Prescription Drug User Fee Act (PDUFA) target action date for the AMX0035 NDA to Sept. 29, 2022, from the original date of June 29, 2022. At the first advisory committee meeting in March 2022 in which six of the 10 members voted no on the question of whether the open-label extension of the phase 2 CENTAUR trial established a conclusion that AMX0035 is effective in the treatment of patients with ALS.