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Setmelanotide

[A224449] Earlier attempts at agonizing MC4R (such as LY2112688) lead to successful weight loss, but also an increase in blood pressure and heart rate. … [A224449] Imcivree was granted EMA orphan designation on November 19, 2018 [L24559] and FDA approval on November 25, 2020.[L24429] On May 4, 2023, it was approved by Health Canada.[L46327]

Pegvaliase

Phenylketonuria is a rare autosomal recessive disorder that is characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH) [A33284] and affects about 1 in 10,000 to 15,000 people in the United … The presence of a PEG moiety in pegvaliase-pqpz allows a reduced immune response and improved pharmacodynamic stability [A33284].

Luspatercept

[A187829,L42455] It was first approved for use in the United States in November 2019 under the brand name Reblozyl® for the treatment of anemia in patients with beta thalassemia who require regular blood … [L42455] Luspatercept is novel in that it ameliorates anemia via action on late-stage erythropoiesis, in contrast to typical erythropoiesis-stimulating agents (ESAs), such as [darbepoetin alfa] and [epoetin

Benralizumab

It is an afucosylated IgG which gives it high affinity for the FcγRIIIα receptor in natural killer cells, macrophages and neutrophils. … [A31293] Benralizumab was FDA approved on November 14, 2017, and was developed by MedImmune, AstraZeneca's global biologic research and development arm.[L1019]

Dabrafenib

[L41955] In May 2018, Tafinlar (dabrafenib), in combination with Mekinist ([DB08911]), was approved to treat anaplastic thyroid cancer caused by an abnormal BRAF V600E gene.[L41955] … It was approved on May 29, 2013, for the treatment of melanoma with V600E or V6000K mutation.[L41955] It was also used for metastatic non-small cell lung cancer with the same mutation.

Netarsudil

humour outflow to act as an inhibitor to the rho kinase and norepinephrine transporters found there as opposed to affecting protaglandin F2-alpha analog like mechanisms in the unconventional uveoscleral … production of aqueous As of December 18, 2017 the FDA approved Aerie Pharmaceutical's Rhopressa (netarsudil ophthalmic solution) 0.02% for the indication of reducing elevated intraocular pressure in

Tirbanibulin

Tirbanibulin exhibits antitumour effects in vitro and in vivo [A225791] and has been investigated for its antitumor efficacy in the management of various cancers, such as prostate cancer and breast cancer … [A225726] On December 14, 2020, tirbanibulin was approved by the FDA for the topical treatment of actinic keratosis on the face or scalp. It is marketed under the brand name Klisyri.

Influenza B virus B/Utah/9/2014 antigen (MDCK cell derived, propiolactone inactivated)

Inactivated virus cannot replicate, and therefore cannot cause disease from infection, even in immunocompromised individuals. … In contrast, live vaccines are produced from "wild-type" or disease-causing viruses that have been attenuated, or weakened, through various laboratory techniques.

Mirdametinib

[L52485] NF1 is an autosomal-dominant genetic condition caused by loss-of-function variants in the _NF1_ tumour suppressor gene,[A265065,A265070] leading to neurofibromin dysfunction and aberrant activation … On February 11, 2025, mirdametinib was approved by the FDA for the treatment of neurofibromatosis type 1 (NF1).

Streptococcus pneumoniae type 6a capsular polysaccharide antigen