Medical Technology

Voxelotor for Sickle Cell Anomaly It is now down to 4-year-olds.

Voxelotor to treat hemolytic anemia in sickle cell disease is now available for children from as young as 4. It’s also available in a tablet that is child-friendly and grape-flavored to administer oral suspensions, making it more accessible.

The indication was previously for patients aged 12 years and over, FDA said in an announcement.

Voxelotor was approved in November 2019 for sickle cell disease. It was described as the first drug that directly blocks sickle hemoglobin polymerization. It binds and stabilizes hemoglobin, stopping the red blood cell from dying and becoming sick.

The FDA approved the new indication for use in children below 4 years of age based on data from a phase 2, that involved 45 children aged between 4 and 11 years old. The FDA reported that 36% of the children had hemoglobin levels greater than 1 g/dL at week 24.

“Complications of sickle cell disease that could cause irreversible organ damage have been known to develop within the first few years following birth,” Ted Love, MD, president, and CEO of Global Blood Therapeutics, stated in a press release.

The company is currently studying voxelotor in infants as young as 9 months old.

FDA granted the agent an expedited approval. Thus, FDA will continue to grant approval to the agent pending additional data that proves that hemoglobin increases have clinical benefits.

Voxelotor is now available in tablets of 500 mg and tablets for oral suspensions of 300 mg. Dosing for ages 12 years and up is 1500 mg once daily. Dosing for children ages 4 to up to 12 years old is based on weight.

The most frequent side effects are headache, vomiting, diarrhea, abdominal pain, nausea, the rash, and fever.

M. Alexander Otto is a physician assistant with a master’s level in medical science. He is also an award-winning medical journalist who has worked for various major news organizations prior to joining Medscape. He is an MIT Knight Science Journalism fellow. Email: [email protected]

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