September 2025: new drugs on the world markets

From September 15 to September 22, the US Food and Drug Administration (FDA) approved three new drugs.  

Enbumyst (bumetanide) is a spray from Corstasis Therapeutics, designed to treat edema in heart failure, as well as liver and kidney diseases. This is the first registered diuretic in the United States in 42 years. Bumetanide is the active ingredient of this drug and has been used in medicine since 1983.  Bumetanide belongs to the class of loop diuretics. To date, bumetanide preparations have been produced in the form of tablets and solutions for intravenous injection.  

It is worth noting that according to studies involving 68 people, Enbumyst is absorbed 33% better than tablets, and the intensity of the diuretic effect is not inferior to the effect of the intravenous form. At the same time, the drug in the form of a spray showed better tolerability and a better safety profile compared to other dosage forms.

Sales of this drug are planned to begin before the end of the year.

The second approved drug is called Keytruda Qlex (pembrolizumab and berahyaluronidase alpha) from MSD (Merck & Co. — in the USA and Canada) and is intended for subcutaneous use in adults with most solid tumors.   

Pembrolizumab is a monoclonal antibody that blocks the programmed death-1 (PD-1) receptor on the surface of immune cells, helping them recognize and attack tumor cells; berahyaluronidase alpha is an enzyme that breaks down hyaluronic acid in tissues, facilitating the penetration and absorption of pembrolizumab when administered subcutaneously.

Subcutaneous administration of the drug provides faster and more convenient use.  At the same time, clinical studies have shown a comparable efficacy profile of the dosage form for subcutaneous and intravenous administration.

The release of Keytruda Qlex is scheduled for the second half of 2025.

The injectable drug Forzinity (elamipretide) from Stealth BioTherapeutics was approved on an expedited basis.  This is the first drug designed to treat Barth syndrome in patients with a body weight of at least 30 kg.  

Barth syndrome is a rare, life—threatening disease of mitochondria (energy components of cells), which usually begins with severe heart failure in infancy and leads to premature death. It is more common in men.

The principle of action of the drug is as follows: it binds to the inside of the mitochondria, improving their structure and function, thereby the cells produce more energy for the normal functioning of the body.

The drug is administered subcutaneously (under the skin) once a day.