The last quarter of 2023 might be fruitful for the life sciences industry in the USA as the Food and Drug Administration (FDA) is going to make a slew of important decisions. The regulator is all set to make a decision about a new genetic treatment, based on the revolutionary CRISPR treatment, for sickle cell disease. The CRISPR-based treatment would be the first such medical intervention to be cleared in the US.
The regulatory authority might also approve the expanded usage of multiple myeloma cell therapy and give a green signal to Amgen’s lung cancer drug, Lumakras. Let us learn about the key decisions that FDA might take this quarter in detail.
FDA to take a call on 2 sickle cell disease medicines
In what can be called positive news to people with sickle cell disease, FDA is all set to make a decision on Exa-cel and Lovo-cel, the two new revolutionary medicines for the deadly disease in December 2023. The victims of sickle cell disease go through frequents bouts of intense pain as their red blood cells deform into rigid sickle-shapes, resulting in clogged blood flow. Currently under review by the US regulator, the two medicines can open a new chapter in the journey of the disease as they are expected to stop or alleviate the pain in the patients for years.
While the decision on Exa-cel will be made on December 8, 2023, the FDA will announce its decision on Lovo-cel by December 20, 2023. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Exa-cel treatment involves gene editing of a patient’s stem cells in order to produce high quantities of a substitute form of the hemoglobin protein. If approved, this would be the first CRISPR-based medicine to be cleared in the USA. On the other hand, Bluebird Bio’s Lovo-cel instructs the cells to make functional protein by adding an engineered form of a hemoglobin gene in the patients’ stem cells.
Expanded Clearance for Bristol Myers’ Multiple Myeloma Therapy
FDA is also expected to come to a decision by December 16 on the approval of the personalized therapy of the multiple myeloma cell treatment Abecma. Bristol Myers Squibb and 2Seventy Bio were the first to market the CAR-T treatment, with them obtaining approval in March 2021. But there is a catch. The treatment can be administered only to patients who received four previous multiple myeloma medicines. Results from the clinical testing showed that Abecma held back the cancer in patients for longer periods of time in comparison to the standard drug regimens.
Amgen’s Lumakras Might Receive Full FDA Approval
Amgen’s novel lung cancer drug Lumakras might receive full approval from the FDA soon. The first-of-its-kind cancer drug has received an expedited clearance in May 2021. However, the regulatory authority questioned the efficacy of the drug at the advisory committee meeting on October 5. When Lumakras was approved, FDA instructed Amgen to conduct follow-up trials to confirm whether it can prevent cancer growth and extend patient life besides shrinking tumors.
Though Lumakras garnered positive results in the confirmatory Phase III trial, the outcomes were not in favor of the survival rate in patients when compared with patients who received chemotherapy. The FDA is expected to make a final decision on Lumakras’ full approval by December 24.
Pfizer’s Etrasimod Approval Expected Soon
The FDA is also expected to clear an experimental drug named Etrasimod from Pfizer in late 2023. Results from the two late-stage clinical studies showed that the treatment can enhance remission rates in patients suffering with ulcerative colitis. The pharma giant is waiting to hear from the FDA with regards to the approval.
The experimental drug is part of Pfizer’s $6.7 billion acquisition of Arena Pharmaceuticals and FDA clearance can fast-track the company’s plan to test the drug’s efficacy in treating conditions such as Crohn’s disease, Alopecia Areata and Eczema. Etrasimod will compete with Bristol Myers’ Zeposia if FDA clears it.
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