A Preview of Potentially Historic FDA Approvals for 2023


The later part of 2023 will see decisions on drugs in four areas such as advanced melanoma, sickle cell disease, Alzheimer’s disease, and depression. BioSpace shines the spotlight on nine drugs to watch towards the end of 2023.

Alzheimer’s Disease

The FDA is considering whether it will grant full approval to Biogen and Esai’s Leqembi. The drug was approved in the first half of 2023 but with the condition that it be used within a clinical study. The CMS has since indicated that it may grant approval to Leqembi but again with conditions. It is also anticipated that the FDA will fully approve Leqembi.

Zuranolone for Postpartum Depression(PPD) and Major Depressive Disorder (MDD)

 By mid-2023 Sage and Biogen will be informed as to whether Zuranolone receives approval and is recognized as the first therapy classified as fast acting in the treatment of MDD and PDD.

Zuranolone is a monoclonal antibody included in a group known to prevent beta-amyloid from forming clumps of plaque. They bind to a receptor and change the receptor’s response to stimuli.

Biogen states that there has been evidence of sustained improvement in symptoms of depression in clinical studies.

Tecentriq for Non-Small Cell Lung Cancer

Roche’s Tecentriq is injectible (subcutaneous). Its formulation is similar to its highly successful anti-PD-(L) 1 therapy. Roche stated that Tencentriq reduced treatment time for patients with advanced non-small cell lung cancer (NSCLC) to minutes rather than one hour when compared to an IV infusion.

ATTR Cardiomyopathy (ATTR-CM) and Polyneuropathy

 Alnylam’s patisiran is scheduled for the FDA’s supplemental approval in October of this year. ATTR is a protein circulating in the bloodstream that becomes misshapen. It builds up in the nerves, heart, and other organs.

Astra and Ionis’ eplontersen reduces the production of the TTR protein and treats both hereditary and non-hereditary ATTR polyneuropathy. It will be reviewed in December of this year.

Lastly, Anylam’s patisiran received approval in 2018 for the treatment of ATTRv-PN. Patisiran showed favorable effects in comparison to placebo during a 12-month follow-up.

Lifileucel to Treat Advanced Melanoma

Although therapies with tumor-infiltrating lymphocytes (TIL) are gaining momentum, the FDA has not yet approved the aforementioned at any level to treat melanoma. TILs are experimental therapies that are developed against various solid tumors.

The FDA is slated to render a November decision on lifileucel developed by Iovance Biotherapeutics to treat advanced melanoma. The C-144-01 trial showcased lifileucel’s impressive performance. During a three-year follow-up, the drug showed a 31 percent ORR.

Sickle Cell Disease

With the focus on rare diseases increasing, it is possible that patients with sickle cell disease (SCD) may be given two new treatment options this year.

The FDA is going to review the merits of CRISPR Therapeutics and Vertex’exagamglogene autotemcel (exa-cel).

Exa-cel is a one-dose treatment designed to treat SCD and the type of beta thalassemia that is transfusion dependent.

The BLAs for exa-cel in SCD and beta-thalassemia (transfusion-dependent) became the first of its kind that was accepted by the FDA for its review. The actionable date is the 30th of March 2024.

These diseases are the result of mutations in the beta-globin gene that harbor a protein essential to hemoglobin function. The mutations cause abnormal hemoglobin and thus anemia in both diseases.

The Exa-cel therapy uses the CRISPR-Cas9 system which edits the patient’s stem cells producing high levels of fetal hemoglobin. Data was presented in June 2022 to the EU Hematology Congress that showed exa-cel preventing and/or substantially reducing TDT transfusions and vaso-occlusive (sickle cell) incidents for over thirty months.

A vaso occlusive crisis occurs when blood flow is blocked by sickled red blood cells and tissues are deprived of oxygen causing an inflammatory response.

Lovotibeglogene autotemcel (lovo-cel)

Lovo-cel is also a one-time gene therapy designed to add copies of modified beta-globin genes into the patient’s hematopoietic stem cells. 36 patients were followed for approximately 32 months. Two patients were followed for eighteen months. Safety data from fifty patients was also included in the results.

The FDA is scheduled to make its decision regarding lovo-cel on December 20, 2023.



Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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