The French National Agency for Medicines and Health Products Safety, also known as ANSM, has granted Temporary Authorization of Use of berotralstat for those living with hereditary angioedema (HAE) above the age of twelve. The drug has not yet received approval from the European Commission (EC), but it addresses a severe unmet medical need and has demonstrated a favorable safety and tolerability profile.
Hereditary angioedema (HAE) is a rare, genetic condition that is characterized by episodes of severe swelling of the blood vessels. While these attacks can be triggered by things like stress or injuries, they often occur without cause. Regardless of reason, symptoms include swelling of the extremities, facial swelling to the point where the eyes swell shut, severe abdominal swelling that can cause vomiting, nausea, and pain, and swelling in the throat that can make it difficult or impossible to breathe. These symptoms are the result of a genetic mutation on chromosome 11 that are inherited in an autosomal dominant pattern. When this mutation appears, a protein called C-1 inhibitor, is unable to properly do its job. This leads to an excess of a peptide called bradykinin, which causes inflammation throughout the body.
ANSM grants this specialized access to drugs that are already presumed to be safe and tolerable through previous research that are indicated for the treatment of severe conditions facing an unmet medical need. Patients treated under this rule are monitored throughout treatment to ensure that everything goes in accordance with the rules.
This decision allows patients to access the care that they need despite the fact that the drug has not yet received proper approval. In berotralstat’s case, approval may come soon. CHMP, which stands for Committee for Medicinal Products for Human Use and is an agency of the European Medicines Agency, has recommended the treatment for approval. They announced at the end of February that berotralastat should be approved for the prevention of attacks common to HAE in patients 12 years old or older.
The final decision on approval is expected in the second quarter of the year. Find the source article here.