Besremi (ropeginterferon alfa-2b-njft) for Polycythemia Vera
Besremi, an interferon alfa-2b, is the first FDA-approved medication for polycythemia vera, a rare blood disease that causes the overproduction of red blood cells. The approval was based on data from the open-label, prospective, phase 1/2 PEGINVERA study (ClinicalTrials.gov Identifier: NCT01193699), which evaluated the efficacy and safety of Besremi in 51 adults with polycythemia vera for 7.5 years. Besremi is administered subcutaneously once every 2 weeks.
Fyarro (sirolimus protein-bound particles [albumin-bound]) for Malignant Perivascular Epithelioid Cell Tumor (PEComa)
Fyarro is an mTOR inhibitor and the first FDA-approved treatment for locally advanced unresectable or metastatic malignant PEComa. The approval was based on data from the multicenter phase 2 AMPECT study (ClinicalTrials.gov Identifier: NCT02494570), which evaluated the efficacy of Fyarro in 34 adults aged 18 years and older with advanced malignant PEComa. The intravenous treatment is expected to be available in the first quarter of 2022.
Livtencity (maribavir) for Post-Transplant Cytomegalovirus Infection/Disease
Livtencity, a cytomegalovirus (CMV) pUL97 kinase inhibitor, is the first drug approved to treat adults and pediatric patients (12 years of age and older and weighing at least 35kg) with post-transplant CMV infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir or foscarnet. The approval was based on data from the phase 3 SOLSTICE trial (ClinicalTrials.gov: NCT02931539), which assessed the efficacy and safety of Livtencity in 352 patients who were recipients of hematopoietic stem cell or solid organ transplant and were refractory or resistant to prior anti-CMV treatment. The drug is available in an oral tablet formulation.
Voxzogo (vosoritide) for Achondroplasia
Voxzogo, a C-type natriuretic peptide analogue, is the first FDA-approved treatment for increasing linear growth in patients 5 years of age and older with achondroplasia and open epiphyses, the most common form of dwarfism. The approval was based on data from a randomized, double-blind, placebo-controlled, phase 3 study (ClinicalTrials.gov Identifier: NCT03197766) that evaluated the efficacy and safety of Voxzogo in 121 children aged 5 to 14.9 years with genetically-confirmed achondroplasia. The treatment is administered by subcutaneous injection.
Vuity (pilocarpine HCI ophthalmic solution) for Presbyopia
Vuity is a cholinergic muscarinic receptor agonist designed to improve near and intermediate vision without impacting distance vision. It is the first FDA-approved eye drop to treat presbyopia, a progressive eye condition. The approval was based on data from the phase 3 GEMINI 1 (ClinicalTrials.gov Identifier: NCT03804268) and GEMINI 2 (ClinicalTrials.gov Identifier: NCT03857542) studies that evaluated the efficacy and safety of Vuity in a total of 750 patients aged 40 to 55 years with presbyopia.
- Besremi. Package insert. PharmaEssentia Corporation; 2021. https://us.pharmaessentia.com/wp-content/uploads/2021/11/BESREMi-USPI-November-2021-1.pdf.
- Fyarro. Package insert. Aadi Bioscience; 2021. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213312lbl.pdf.
- Livtencity. Package insert. Takeda Pharmaceutical Company Limited; 2021. https://content.takeda.com/?contenttype=pi&product=liv&language=eng&country=usa&documentnumber=1.
- Voxzogo. Package insert. BioMarin Pharmaceutical Inc.; 2021. https://pages.bmrn.com/rs/424-CBN-212/images/prescribing_information.pdf.
- Vuity. Package insert. AbbVie; 2021. https://www.rxabbvie.com/pdf/vuity_pi.pdf.