54 steroidal small molecules approved by the FDA

54 steroidal small molecules approved by the FDA are a class of biologically active polycyclic compounds composed of four fused rings arranged as three cyclohexane rings (A, B, and C) and one cyclopentane ring (D). These rings are known as steranes (cyclopentanephydrophenanthrene). Steroids are lipophilic, low-molecular-weight compounds biosynthesized from cholesterol and also obtained from various marine and terrestrial sources. The steroid family includes sterols (cholesterol), hormones (gonadotropins and adrenocortical hormones), bile acids, and their metabolites. All of these play important roles in the physiological and biochemical functions of organisms.

---

Steroids are not only synthetic but also occur naturally in the human body. They include:

i) Sex hormones, including androgens, estrogens, and progestins, which are responsible for sexual differentiation and reproduction; ii) Corticosteroids, such as glucocorticoids (which regulate metabolism and immune function) and mineralocorticoids (which maintain blood volume and electrolyte levels);

iii) Anabolic steroids, which help increase muscle mass and promote bone building.

Between 2000 and 2024, the US FDA approved as many as 1,410 new molecular entities (NMEs). Of these, 54 are steroidal NMEs and have been approved for the treatment of a variety of diseases, such as asthma, cancer, inflammation, menopausal symptoms, skin problems, and neurodegenerative diseases.

Next, we will introduce the steroid drugs that have been launched on the market:

Vamorolone, trade name: Agamree; indication: Duchenne muscular dystrophy, approved for marketing in 2023.

Zuranolone (trade name: Zulresso). Indication: Postpartum depression. Approved for marketing in 2023.

Omaveloxolone (trade name: Skyclarys); Indication: Friedreich’s ataxia, a hereditary spinal cord disorder characterized by motor incoordination, difficulty walking, and speech impairment, often occurring in children and adolescents. Approved for marketing: 2023.

Taurursodiol/sodium phenylbutyrate, trade name: Relyvrio; indication: amyotrophic lateral sclerosis (ALS), approved for marketing in 2022.

Ganaxolone, trade name: Ztalmy; indications: epilepsy or epileptic seizures, approved for marketing in 2022.

Drospirenone/estetrol (Drospirenone/estetrol), trade name: Nextstellis; indication: contraceptive pill, approved for marketing in 2021.

Fluoroestradiol F18 (trade name: Cerianna); indication: diagnostic imaging agent for certain breast cancer patients, approved for marketing in 2020.

Clascoterone, trade name: Winlevi; indication: acne, approved for marketing in 2020.

Brexanolone, trade name: Zulresso; indication: postpartum depression, approved for marketing in 2019.

Segesterone acetate/ethinyl estradiol (trade name: Annovera); indication: contraceptive pill, approved for marketing in 2018.

Deflazacort, trade name: Emflaza; indication: Duchenne muscular dystrophy (DMD), approved for marketing in 2017.

Obeticholic acid (trade name: Ocaliva); indications: rare chronic liver diseases (such as acute hepatic porphyria AHP and autoimmune hepatitis AIH), approved for marketing in 2016.

Cholic acid, trade name: Cholbam; indication: bile acid synthesis disorders (BASDs), approved for marketing in 2015.

Deoxycholic acid (trade name: Kybella); Indications: Improves the appearance and morphology of moderate to severe fat under the chin (submental fat). Approved for marketing in 2013.

Abiraterone acetate (trade name: Zytiga); indication: used in combination with prednisone to treat prostate cancer, approved for marketing in 2011.

Estrone sulfate (sodium)/bazedoxifene, trade name: Duavee; indication: moderate to severe hot flashes, approved for marketing in 2013.

Ulipristal, trade name: Ella; indication: emergency contraceptive, approved for marketing in 2010.

Estradiol valerate/dienogest (trade name: Natazia); indication: contraceptive pill, approved for marketing in 2010.

Flunisolide, trade name: Aerospan; indication: allergic rhinitis, approved for marketing in 2009.

Levonorgestrel (trade name: Skyla); indication: contraceptive pill, approved for marketing in 2009.

Ethinylestradiol/levonorgestrel (trade name: Lybrel); indication: contraceptive pill, approved for marketing in 2007.

Calcipotriene/betamethasone dipropionate, trade name: Taclonex; indication: psoriasis vulgaris, approved for marketing in 2008.

Triamcinolone acetonide, trade name: Trivaris; indications: uveitis and inflammatory ocular diseases, approved for marketing in 2008.

Difluprednate, trade name: Durezol; Indications: Treatment of eye swelling and pain after eye surgery, approved for marketing in 2008.

Equilin sodium sulfate, trade name: Cenestin; indications: menopausal symptoms (such as hot flashes, vaginal dryness), approved for marketing in 2008.

Drospirenone/ethinyl estradiol (trade name: Yaz); indications: oral contraceptives, premenstrual dysphoric disorder (PMDD) and acne, approved for marketing in 2007.

Clobetasol propionate, trade name: Olux E; indications: eczema and psoriasis, approved for marketing in 2007.

Hydrocortisone butyrate, trade name: Locoid; indications: eczema, dermatitis, allergies and rash, approved for marketing in 2007.

Ciclesonide, trade name: Omnaris; Indications: Asthma in adults and children aged 12 years and above, approved for marketing in 2006.

Fluticasone propionate, trade name: Advair HFA; indications: eczema and psoriasis, approved for marketing in 2006.

Fluticasone furoate (trade name: Veramyst); indication: allergic and non-allergic nasal diseases (such as rhinitis); approved for marketing in 2007.

Fluticasone furoate (trade name: Breo Ellipta); indication: chronic obstructive pulmonary disease (COPD, including chronic bronchitis and/or emphysema); approved for marketing in 2013.

Prednisolone sodium phosphate, trade name: Orapred ODT; indications: arthritis, blood and immune diseases, skin and eye problems, respiratory problems, cancer and severe allergies, approved for marketing in 2006.

Budesonide, trade name: Pulmicort Flexhaler; indications: dyspnea, chest tightness, wheezing and cough caused by asthma, approved for marketing in 2006.

Etonogestrel (trade name: Implanon); indication: contraceptive pill, approved for marketing in 2006.

Desonide, trade name: Verdeso; indications: eczema, dermatitis, allergies and rash, approved for marketing in 2006.

Ciclesonide, trade name: Omnaris; indications: asthma and allergic rhinitis, approved for marketing in 2006.

Fluocinonide, trade name: Vanos; Indications: Treatment of skin diseases such as eczema and seborrheic dermatitis, approved for marketing in 2006.

Fluocinolone acetonide, trade name: Retisert; indications: eczema, dermatitis, allergies, rash, approved for marketing in 2004.

Medroxyprogesterone acetate (trade name: Prempro/Premphase); indications: menopausal symptoms (such as hot flashes, vaginal dryness), approved for marketing in 2003.

Clobetasol propionate (trade name: Clobex); indications: skin inflammation and itching, approved for marketing in 2004.

Estradiol hemihydrate, trade name: Estrasorb; Indications: Contraceptive pills, approved for marketing in 2003.

Eplerenone, trade name: Eplerenone; indications: hypertension and congestive heart failure (CHF), approved for marketing in 2002.

Dutasteride, trade name: Avodart; indication: benign prostatic hyperplasia, approved for marketing in 2001.

Norethindrone acetate (trade name: Activella); indications: menopausal symptoms (such as hot flashes and vaginal changes), approved for marketing in 2001.

Norelgestromin/ethinyl estradiol, trade name: Ortho Evra; indication: contraceptive, approved for marketing in 2001.

Cortisone acetate, trade name: Cortone; indications: allergic diseases, skin conditions, ulcerative colitis, arthritis, lupus, psoriasis and respiratory disorders, approved for marketing in 2001.

Norethindrone acetate (trade name: Activella); indications: menopausal symptoms (such as hot flashes and vaginal changes), approved for marketing in 2001.

Fluticasone propionate, trade name: Advair Diskus 500/50; indication: inflammation, approved for marketing in 2000.

Fulvestrant, trade name: Faslodex; indication: metastatic breast cancer, approved for marketing in 2002.

Beclomethasone dipropionate, trade name: Qvar 80; indications: psoriasis, eczema, dermatitis, allergies, rash, approved for marketing in 2000.

Estradiol cypionate, trade name: Lunelle; indications: menopausal symptoms, approved for marketing in 2000.

Betamethasone dipropionate/clotrimazole (trade name: Lotrisone); indications: fungal infections of the feet, groin, and body in people aged 17 years and older. Approved for marketing in 2000.

Desogestrel/ethinyl estradiol (trade name: Cyclessa); indication: combined hormonal drug (for contraception), approved for marketing in 2000.