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FDA Updates for the Week of May 2, 2022


The FDA has approved a rare disease therapy and an additional indication for Enhertu, and grants priority review for Imfinzi in biliary tract cancer. Regulators have issued complete response letters for three therapies. The agency also has scheduled an advisory committee for Nuplazid in Alzheimer’s psychosis.

FDA approves Enhertu for earlier use in metastatic breast cancer.

The FDA has approved Enhertu (fam-trastuzumab deruxtecan-nxki) to treat adults with unresectable or metastatic HER2 positive breast cancer who have received a prior anti-HER2 therapy.

Developed by Daiichi Sankyo and AstraZeneca, Enhertu’s new approval was based on positive results from the pivotal DESTINY-Breast03 phase 3 trial that showed Enhertu reduced the risk of disease progression or death by 72% versus Kadcyla (trastuzumab emtansine) in patients previously treated with trastuzumab and a taxane.

The previously granted accelerated approval of Enhertu in later line HER2 positive metastatic breast cancer is now converted to regular approval.

FDA approves Qelbree for ADHD in adults.

The FDA has approved the supplemental new drug application for Qelbree (viloxazine) to treat adults with attention-deficit hyperactivity disorder (ADHD). It was approved in April 2021 to children 6 to 17 years of age.

Developed by Supernus Pharmaceuticals, Qelbree’s applications for adult was supported by data from positive results in a phase 3 study. That trial showed that Qelbree at a daily dose of up to 600 mg met the primary endpoint in improving the symptoms of ADHD from baseline to end of study as measured by ADHD Investigator Symptom Rating Scale.

Qelbree, novel non-stimulant treatment for children with ADHD in a decade, was launched in the second quarter of 2021 and for the year, closed the year with 13,380 prescriptions written, according to the company.

FDA approves Cuvrior for rare disease.

The FDA has approved Cuvrior, (trientine tetrahydrochloride) for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. Penicillamine is currently approved as a first-line treatment of Wilson’s disease in the United States with about one third of patients developing intolerance. Orphalan plans to launch Cuvrior in early 2023.

Wilson disease is a rare, genetic disorder characterize excess copper in the liver, brain and corneas, according to the National Organization for Rare Diseases. If left untreated, it can cause liver disease, central nervous system dysfunction, and death.

AstraZeneca’s blockbuster cancer drug granted priority review for rare cancers.

The FDA has granted priority review for AstraZeneca’s supplemental biologics license application (sBLA) for Imfinzi (durvalumab), in combination with standard-of-care chemotherapy, for locally advanced or metastatic biliary tract cancer (BTC).

Imfinzi, a human monoclonal antibody, is already cleared to treat several other cancers, including unresectable, Stage 3 non-small cell lung cancer (NSCLC), extensive-stage small cell lung cancer, and advanced bladder cancer, AstraZeneca said in a news release.

The FDA’s Prescription Drug User Fee Act action date for its regulatory decision for Imfinzi for BTC is during the third quarter of 2022.

FDA issues CRL for surufatinib for rare pancreatic cancer.

The FDA has issued a complete response letter for surufatinib for the treatment of patients with pancreatic and non-pancreatic neuroendocrine tumors (NETs). The FDA indicated that the application, based on two positive phase 3 trials in China and one bridging study in the United States, does not support an approval at this time.

The CRL indicated that a multi-regional clinical trial is required for U.S. approval. The agency would like to see a trial that includes that includes patients who are more representative of the U.S. patient population and aligned to current U.S. medical practice. In addition, pandemic-related issues concerning inspection scheduling and access contributed to the FDA action. This action by the FDA is not related to any safety issues with surufatinib, the company said.

Developed by China-based Hutchmed, surufatinib is an oral inhibitor of angiogenesis and immune modulation. It is angio-immuno kinase inhibitor that inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptors (VEGFR) and fibroblast growth factor receptor (FGFR). The FDA had accepted the new drug application in July 2021.

FDA issues CRL for toripalimab.

The FDA has issued a complete response letter for toripalimab, an anti-PD-1 monoclonal antibody to treat patients with metastatic nasopharyngeal carcinoma. Developed by Coherus BioSciences and Shanghai Biosciences, toripalimab is being studied in combination with gemcitabine and cisplatin for first-line treatment of this cancer.

The regulatory agency has requested a quality process change that the companies believe is readily addressable. The companies plan to meet with the FDA and expect to resubmit the BLA by mid-summer 2022. The agency also communicated in the CRL that the review timeline for the BLA resubmission would be six months because of required onsite inspections have been hindered by travel restrictions related to the COVID-19 pandemic in China.

FDA issues CRL for migraine therapy.

The FDA has issued a complete response letter for Axsome Therapeutics’ meloxicam-rizatriptan combination therapy (AXS-07) for the acute treatment of migraine. The principal reasons given in the CRL relate to chemistry, manufacturing, and controls (CMC) considerations. The CRL identified the need for additional CMC data related to the drug product and manufacturing process.

Axsome officials believe the issues raised in the CRL are addressable and they intend to provide potential timing for a resubmission following consultation with the FDA. No clinical efficacy or safety issues were raised, and no additional clinical studies are required by the FDA to support approval.

FDA schedules advisory committee for Nuplazid in Alzheimer’s psychosis.

The FDA’s Psychopharmacologic Drugs Advisory Committee will meet on June 17, 2022, to review Acadia Pharmaceuticals’ resubmitted supplemental new drug application (sNDA) for Nuplazid (pimavanserin) for the treatment of hallucinations and delusions associated with Alzheimer’s disease psychosis. The target action date for the application is August 4, 2022.

The agency had issued a complete response letter in April 2021 for this therapy to treat dementia-related psychosis, saying that in the HARMONY study there was a lack of statistical significance in some of the subgroups of patients with dementia. In addition, FDA officials said there was a lack of sufficient number of patients in dementia subtypes. Regulatory authorities also said that it considered the phase 2 Study-019 not adequate because it was not well controlled and because protocol deviations occurred. No safety issues were raised in the response letter.

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