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hydroxyprogesterone caproate (Makena SQ / 17P / Makena)

✓ Approved

Lumara Health · PGR

What is hydroxyprogesterone caproate?

hydroxyprogesterone caproate is a therapeutic agent developed by Lumara Health. It is approved for therapeutic indications via injectable (others) or intramuscular (im) injection or subcutaneous injection.

Drug Profile

Brand NamesMakena SQ, 17P, Makena
CompanyLumara Health
Molecular TargetPGR
RouteInjectable (Others), Intramuscular (IM) Injection, Subcutaneous Injection
StatusApproved
Sources: ClinicalTrials.gov, Lumara Health

Mechanism of Action

Molecular Targets

hydroxyprogesterone caproate acts on 1 molecular target:

PGRprogesterone receptor (NR3C3, PR)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

hydroxyprogesterone caproate is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Pregnancy, puerperium and perinatal conditionsPremature labour✓ Approved

Related Research Articles

PubMedFrontiers in endocrinology2026-07-25

Stress-associated transient enlargement of pre-existing adrenal hyperplasia in 21-hydroxylase deficiency: a case report.

Zheng Chengcheng C, Zhao Lianling L, Chen Tao T

The adrenal gland plays a pivotal role in the stress response via the hypothalamic-pituitary-adrenal (HPA) axis and the sympatho-adrenomedullary system (SAMS). Congenital adrenal hyperplasia (CAH) results from pathogenic variants in genes encoding adrenal steroidogenic enzymes. The most common form, 21-hydroxylase deficiency, impairs cortisol biosynthesis, leading to compensatory overproduction of adrenocorticotropic hormone (ACTH) and chronic adrenal hyperplasia. A 40-year-old man was referred for incidentally detected bilateral adrenal lesions on abdominal computed tomography (CT). CT revealed marked bilateral adrenal enlargement with clear contours. One month before admission, he had an acute upper respiratory tract infection and recovered after treatment with a cephalosporin. On admission, physical examination showed hyperpigmentation of the nipples region, lips, gums, and buccal mucosa. Superficial lymph nodes were palpable. He reported a history of infertility and suspected precocious puberty. Endocrinology markers demonstrated an elevated ACTH level (180 ng/L; reference range 5.0-78.0 ng/L) with a cortisol level of 174 nmol/L (reference range 133.0-537.0 nmol/L). Luteinizing hormone and follicle-stimulating hormone levels were both low, whereas dehydroepiandrosterone sulfate was elevated. Markedly elevated 17α-hydroxyprogesterone levels and a blunted cortisol response were observed under ACTH-stimulated conditions. Based on the clinical phenotype and biochemical findings, CAH due to 21-hydroxylase deficiency was diagnosed and considered consistent with the simple virilizing form, which was subsequently genetically supported by the CYP21A2 I172N homozygous mutation in exon 4. Remarkably, without any specific treatment, adrenal volume decreased substantially within one month, from 61.06 cm3 to 33.47 cm3, corresponding to an approximately 1.8-fold reduction, although the adrenal glands remained enlarged compared with normal reference values. After excluding adrenal-related tumors, hemorrhage, infections and autoimmune diseases, we postulated that the transient morphological changes reflected stress-induced compensatory hypertrophy and/or hyperplasia of the adrenal cortex following the respiratory infection, aimed at augmenting cortisol production to modulate inflammation and maintain homeostasis. In patients with 21-OHD and residual enzymatic activity, infectious stress may trigger transient enlargement of pre-existing adrenal hyperplasia, potentially as a compensatory response to increased cortisol demand, followed by partial regression after stress resolution.

PMID 42500133
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PubMedFrontiers in endocrinology2026-07-25

Clinical, metabolomic, and proteomic profiles associated with reproductive outcomes in unexplained recurrent pregnancy loss.

Song Zicheng Z, Jiang Yishi Y, Zhu Zhixing Z, Che Yan Y et al.

To identify preconception clinical and multi-omics factors associated with conception and early pregnancy loss in women with unexplained recurrent pregnancy loss (URPL). In this prospective cohort study, 149 women with URPL selected from 420 outpatients based on guideline-recommended criteria were enrolled between November 2024 and May 2025 and followed-up for 12 months. Preconception fasting plasma was analyzed for clinical biomarkers, untargeted metabolomics, and data-independent acquisition proteomics. Outcomes included conception, ongoing pregnancy beyond 12 weeks and early pregnancy loss before 12 weeks. Multivariable logistic regression was used to evaluate the associations of clinical and multi-omics factors with reproductive outcomes, adjusting for maternal age, body mass index, number of prior losses, and use of assisted reproductive technology. Of 149 women, 99 conceived (66.4%) during the follow-up. By 12 weeks of gestation, 67 (67.7%) had ongoing pregnancies and 32 (32.3%) experienced early pregnancy loss. Higher testosterone was associated with lower probability of conception (adjusted odds ratio [aOR] 0.50, 95% confidence interval [CI] 0.28-0.89, p = 0.019). Women who conceived showed higher levels of progesterone-related metabolites, including 17-hydroxyprogesterone (fold change, FC = 3.89), pregnanediol 3-O-glucuronide (FC = 2.37), and pregnanetriol 3α-O-β-D-glucuronide (FC = 2.39). Among women who conceived, higher prolactin was associated with higher odds of early pregnancy loss (aOR 1.09, 95% CI 1.01-1.18, p = 0.036), and anti-phosphatidylserine/prothrombin showed a borderline association (aOR 1.07, 95% CI 1.00-1.14, p = 0.052). Early pregnancy loss was characterized by lower bile acid-related metabolites and higher caffeine and methylxanthine metabolites. Proteomic analysis showed enrichment of bile acid biosynthetic process. After adjustment, higher 7α,12α-dihydroxy-3-oxocholest-4-en-27-oic acid was associated with lower odds of early pregnancy loss (aOR 0.64, 95% CI 0.44-0.95, p = 0.025). Higher testosterone was associated with lower odds of conception. Among women who conceived, early pregnancy loss was associated with higher prolactin, borderline higher anti-PS/PT, lower bile acid-related metabolites, and higher caffeine-related metabolites, with 7α,12α-dihydroxy-3-oxocholest-4-en-27-oic acid identified as an exploratory metabolomic candidate. These findings suggest that potential androgen-related biology, prolactin, non-criteria antiphospholipid antibodies, and bile acid metabolism may be associated with reproductive outcomes in URPL, warranting validation in independent cohorts.

PMID 42500125
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PubMedFrontiers in endocrinology2026-07-22

Modified-release hydrocortisone (Efmody®) in children with congenital adrenal hyperplasia: a retrospective registry study.

Lankes Erwin E, Wiebelt Levin L, Helge Kathrin Bettina KB, Schnabel Dirk D et al.

Replacement therapy for congenital adrenal hyperplasia (CAH) in childhood includes hydrocortisone and, in salt-wasting forms, addition of fludrocortisone. Immediate-release hydrocortisone often fails to adequately suppress early morning 17-hydroxyprogesterone (17OHP) levels. Hydrocortisone modified-release capsules (HMRC, Efmody®) were approved by the EMA in 2021 for the treatment of patients aged ≥12 years with CAH. This study evaluates the efficacy and safety of HMRC in children and adolescents based on real-world data collected from the I-CAH registry. A single-center retrospective analysis of children with CAH was conducted. Linear mixed models (LMMs) were used to calculate pre-post comparisons of 17OHP saliva profiles, growth rate, bone age, body mass index, hydrocortisone and fludrocortisone dosage, blood pressure, and safety parameters before and during HMRC therapy (study registration, https://drks.de/search/de, DRKS00038319). The median age of the 36 children with CAH at the time of treatment switching to HMRC was 10 years (interquartile range [IQR]: 8-14 years). Of these, 15 were prepubertal and 21 were (post)pubertal. HMRC therapy was associated with a deceleration of previously increased growth velocity (pre-switch: 0.1 SDS/year, 95% CI: 0.0 to 0.2; post-switch: -0.1 SDS/year, 95% CI: -0.2 to 0.1) and with a reduction of median difference of bone age and chronological age by 0.50 years (IQR -1.17-0.27, range -2.2-2.00). Mean hydrocortisone dose increased by 2.4 mg/m²/day during HMRC treatment. Mean morning 17OHP concentrations significantly decreased from 337 ng/L (95% CI: 240-437) to 214 ng/L (95% CI: 155-294) after switching to HMRC treatment. No adrenal crisis was observed. Twice-daily HMRC therapy can also improve CAH therapy in children and adolescents in terms of hormonal control and growth.

PMID 42483672
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PubMedEuropean journal of pediatrics2026-07-20

Health-related quality of life in children and adolescents with congenital adrenal hyperplasia: a cross-sectional study using the pediatric quality of life inventory.

Kocaay Pınar P, Gürbüz Emre E, Çetin Sirmen Kızılcan SK, Kırgıl Berrak Naz BN et al.

Congenital adrenal hyperplasia (CAH) is a chronic endocrine disorder requiring lifelong management that may significantly impact health-related quality of life (HRQoL) in pediatric patients. Despite growing recognition of psychosocial outcomes in CAH, data from diverse populations remain limited. This cross-sectional study evaluated HRQoL in 40 children and adolescents with CAH (aged 2-18 years) followed at our pediatric endocrinology clinic, compared with 82 healthy controls. The Pediatric Quality of Life Inventory (PedsQL) was administered to assess physical, emotional, social, and school functioning domains. Clinical data including 17-hydroxyprogesterone (17-OHP), adrenocorticotropic hormone (ACTH), androstenedione levels, bone age, treatment regimens, and surgical history were collected. Statistical analyses included Mann-Whitney U test, chi-square test, and Spearman correlation. The mean age of patients with CAH was 11.85 ± 4.3 years. Based on child self-reports, total HRQoL scores were significantly lower in the CAH group compared with controls (81.69 ± 11.6 vs. 87.95 ± 6.0, p = 0.007). Emotional functioning (p = 0.001), social functioning (p = 0.039), school functioning (p = 0.048), and psychosocial health scores (p = 0.002) were also significantly lower in patients, whereas physical functioning scores were comparable between groups (p = 0.117). Parent proxy-reports similarly demonstrated significantly lower total HRQoL (p = 0.001), social functioning (p < 0.001), school functioning (p = 0.010), and psychosocial health scores (p = 0.001) in the CAH group. No significant differences were observed between child and parent assessments. Disease duration showed positive correlations with child-reported physical functioning (r = 0.402, p = 0.012), emotional functioning (r = 0.595, p < 0.001), psychosocial health (r = 0.394, p = 0.014), and total HRQoL scores (r = 0.393, p = 0.015). No consistent associations were identified between HRQoL scores and glucocorticoid dose, BMI SDS, height SDS, biochemical control, or history of surgery. Children and adolescents with CAH experience substantial impairments in HRQoL across all functional domains, particularly in psychosocial functioning.These findings should be interpreted with caution given the relatively small sample size of this study. Nevertheless, they suggest.Nevertheless, they suggest the need for comprehensive, multidisciplinary care approaches that address not only biochemical control but also psychological and social well-being in pediatric CAH management. • Congenital adrenal hyperplasia requires lifelong glucocorticoid therapy. Its potential to impair health-related quality of life in affected children is increasingly recognized. • Children and adolescents with CAH had significantly lower HRQoL than healthy controls on both self- and parent proxy-reports, with impairment concentrated in the psychosocial (emotional, social, and school) domains rather than physical functioning. • HRQoL scores were unrelated to glucocorticoid dose, biochemical control, anthropometry, or surgical history.

PMID 42474542
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PubMedBioresource technology2026-07-17

N-(3-oxohexanoyl)-homoserine lactone-assisted enrichment reshapes functional microbial consortia for chain elongation in electrofermentation.

Qiang Haifeng H, Jing Yimin Y, Xu Xianbao X, Heo Seongbong S et al.

The functional microbial consortia supporting chain elongation determine medium-chain carboxylate recovery from organic wastes, but how signal-molecule-assisted enrichment shapes chain-elongating bacteria (CEB), electroactive bacteria (EAB), and competing guilds in electrofermentation remains unclear. Here, three N-acyl-homoserine lactones: N-butyryl-homoserine lactone (C4-HSL), N-octanoyl-homoserine lactone (C8-HSL), and N-(3-oxohexanoyl)-homoserine lactone (3OC6-HSL), were supplied during microbial enrichment, and the subsequent electrofermentation was conducted fed with sludge fermentation broth. Compared with the Control (without signaling molecules), 3OC6-HSL had the strongest response, increasing caproate production by 94.0%, compared with 16.9% and 27.3% for C4-HSL and C8-HSL, respectively. It also increased the apparent caproate electron transfer efficiency by 20.7 percentage points, increased the abundance of CEB (44.9% vs. 33.2%) and EAB (14.3% vs. 6.6%), and reduced the abundance of homoacetogens (12.1% vs. 33.7%). Co-occurrence network analysis revealed more modular and compact inferred associations, with 25.0% more modules and a 34.7-49.3% shorter average path length. Metagenomic analysis revealed enhanced reverse β-oxidation, QS, chemotaxis, and flagellar assembly potentials, and the expression levels of acetyl-CoA acyltransferase (ACAT/fadA) and acyl-CoA dehydrogenase (ACADS/ACADM) increased by 162.1% and 96.6%, respectively. Clostridium kluyveri dominated the ACAT contribution (85.9%). Overall, enrichment-phase 3OC6-HSL supplementation was associated with a caproate-oriented microbial consortium and improved caproate recovery without continuous signal dosing.

PMID 42462831
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PubMedAlzheimer's research & therapy2026-07-16

Steroid hormones in Alzheimer's disease: a molecular analysis of blood and cerebrospinal fluid.

Muk Tik T, Wretlind Asger A, Hooshmand Kourosh K, Clos-Garcia Marc M et al.

Alzheimer's disease (AD) disproportionately affects women, yet existing studies have been limited to single biofluids, individual hormones, or single dementia subtypes, leaving sex-stratified profiles across the cognitive impairment spectrum poorly defined. Here we address this gap by simultaneously quantifying nine steroid hormones spanning glucocorticoid (cortisol, 11-deoxycortisol), mineralocorticoid (aldosterone), progestogen (progesterone, 17-hydroxyprogesterone), androgen (testosterone, dihydrotestosterone), and estrogen (estradiol, estrone) pathways in paired CSF and plasma across five cognitive categories: no cognitive impairment, mild cognitive impairment (MCI) with AD pathology, MCI without AD pathology, AD dementia, and vascular dementia. Steroid hormones were quantified by liquid chromatography-tandem mass spectrometry in paired cerebrospinal fluid (CSF) and plasma samples drawn from the same individuals from a cross-sectional study including 204 participants across five cognitive categories at the Danish Dementia Research Centre. Gender-stratified generalized linear models adjusted for age were applied. Cortisol findings were validated externally using the Alzheimer's Disease Neuroimaging Initiative (ADNI) cohort (n = 426). Female participants with AD exhibited elevated CSF cortisol (fold change [FC] = 1.13; P = .04) and CSF 11-deoxycortisol (FC = 1.01; P = .03), alongside reduced plasma progesterone (FC = 0.90; P = .04). Male participants with AD showed elevated plasma aldosterone (FC = 1.19; P = 2.81e-03; q = 0.02). CSF cortisol correlated with amyloid-β42 and phosphorylated tau in female participants. ADNI validation confirmed elevated plasma cortisol in AD, with a larger effect in female participants. Gender-dependent steroid hormone dysregulation in dementia highlights cortisol and aldosterone as potentially modifiable targets warranting further investigation.

PMID 42458616
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