FDA Approved Drugs: June, 2024

FDA Approved Drugs: June, 2024

RYTELO

RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration. It is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

It is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron.

IQIRVO

Iqirvo (elafibranor) is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. Iqirvo was in-licensed from GENFIT in 2021. The accelerated approval of Iqirvo is based on data from the Phase III ELATIVE trial published in the New England Journal of Medicine.

SOFDRA

Sofdra (sofpironium) gel, 12.45%, is now the first and only chemical entity approved for excessive underarm sweating (primary axillary hyperhidrosis). The treatment is indicated for usage in adults and children aged nine years and above. The FDA's decision was based on results from the two Phase III CARDIGAN studies in 701 patients.

PIASKY

PiaSky (crovalimab-akkz) is the first monthly subcutaneous (SC) treatment for paroxysmal nocturnal haemoglobinuria (PNH). Additionally, with the option to self-administer, PiaSky may provide an alternative to existing intravenous (IV) C5 inhibitors, potentially helping to reduce treatment burden. The recommendation is based on the COMMODORE 2 study results, where SC PiaSky given every month demonstrated equivalent disease control and comparable safety to IV eculizumab given every two weeks.

OHTUVAYRE

Ohtuvayre (ensifentrine) is the first inhaled therapy for the maintenance treatment of COPD that combines bronchodilator and non-steroidal anti-inflammatory activities in one molecule. Verona has evaluated nebulized Ohtuvayre in its Phase 3 clinical program ENHANCE (�Ensifentrine as a Novel inHAled Nebulized COPD thErapy�) for COPD maintenance treatment. Ohtuvayre met the primary endpoint in both ENHANCE-1 and ENHANCE-2, demonstrating statistically significant and clinically meaningful improvements in lung function. A fixed-dose combination of ensifentrine and glycopyrrolate, a LAMA, is currently under development for the maintenance treatment of COPD. Ensifentrine has potential applications for development in non-cystic fibrosis bronchiectasis, cystic fibrosis, asthma and other respiratory diseases.

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