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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

PubMedJournal of clinical medicine2026-09-15

Clinical, Metabolic and Hormonal Overlap Between Nonclassic Congenital Adrenal Hyperplasia and Polycystic Ovary Syndrome: An Exploratory Study.

Yavuz Arzu A, Coskun Aylin A, Uzman Dilara Tekin DT, Hatipoglu Esra E

Objective: Polycystic ovary syndrome (PCOS) and nonclassic congenital adrenal hyperplasia (NCAH) overlap clinically, but direct comparative data specifically addressing non-National Institutes of Health (non-NIH) Rotterdam phenotypes of polycystic ovary syndrome are limited. We compared women with polycystic ovary syndrome, of whom 66.7% were classified as having non-NIH Rotterdam phenotypes, with women with nonclassic congenital adrenal hyperplasia and healthy controls to characterize the extent of phenotypic overlap and evaluate the discriminatory performance of individual androgen measures. Methods: This single-center, prospective, exploratory cross-sectional study evaluated 58 women divided into three groups: PCOS (n = 24, Rotterdam criteria), NCAH due to 21-hydroxylase deficiency (n = 16, previously confirmed by adrenocorticotropic hormone (ACTH)-stimulated 17-hydroxyprogesterone (17-OHP)), and healthy controls (n = 18). Demographic, clinical, metabolic, and hormonal parameters were compared using analysis of variance (ANOVA), Kruskal-Wallis with Dunn's post hoc test (Bonferroni-adjusted), and chi-square or Fisher's exact tests. A sensitivity analysis was performed after excluding 12 participants receiving oral contraceptives (n = 8) or glucocorticoids (n = 4). Results: Within the PCOS group, 66.7% had non-NIH Rotterdam phenotypes. Hirsutism, defined as a modified Ferriman-Gallwey (mFG) score ≥ 8, was more frequent in both patient groups than in controls (p = 0.003) but did not differ between PCOS and NCAH. Total testosterone levels were 0.40 [0.20-0.60], 0.59 [0.45-0.94], and 0.20 [0.15-0.20] ng/mL in the PCOS, NCAH, and control groups, respectively, and androstenedione levels were 1.40 [1.20-2.20], 2.55 [1.64-4.07], and 0.87 [0.56-1.00] ng/mL, respectively; both were higher in the patient groups than in controls (both p < 0.001), without differing between PCOS and NCAH. Basal 17-OHP was the only parameter distinguishing NCAH from both PCOS and controls (p < 0.001). Dehydroepiandrosterone sulfate (DHEAS) levels were 356.5 [217-422], 292 [156-448], and 219 [146-240] µg/dL, respectively (p = 0.020), with a significant difference only between PCOS and controls. Anti-Müllerian hormone (AMH) levels were 3.27 [3.02-4.13], 3.13 [2.87-3.54], and 3.34 [2.93-5.37] ng/mL, respectively, with no between-group difference (p = 0.600). Other metabolic parameters, gonadotropins, estradiol, and prolactin showed no between-group differences. The principal hormonal findings remained unchanged after excluding women receiving oral contraceptives or glucocorticoids. Conclusions: In this PCOS cohort, in which 66.7% of women were classified as having non-NIH Rotterdam phenotypes, PCOS and NCAH showed substantial clinical, metabolic, and hormonal overlap, whereas basal early-follicular 17-OHP was the only parameter that consistently distinguished NCAH from both PCOS and healthy controls. These findings support the inclusion of basal 17-OHP in the evaluation of women presenting with hyperandrogenism.

PMID 42739527
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PubMedJCEM case reports2026-09-13

Adult nonclassic P450 oxidoreductase deficiency diagnosed 11 years after reproductive-endocrine suspicion.

Kono Takashi T, Hyakutake Saaya S, Nishimura Motoi M, Ishikawa Hiroshi H et al.

Nonclassic P450 oxidoreductase (POR) deficiency (PORD) may remain suspected but unconfirmed in adult women with long-standing menstrual irregularity and infertility when skeletal anomalies, virilization, and overt adrenal insufficiency are absent. We report a 45-year-old Japanese woman in whom PORD had been suspected during infertility care at age 34 because of an inappropriately elevated, nonluteal progesterone value with no marked 17-hydroxyprogesterone elevation, but genetic testing was not pursued. Reevaluation for recurrent abnormal uterine bleeding and a large ovarian cyst prompted 3-point liquid chromatography-tandem mass spectrometry (LC-MS/MS) steroid profiling at baseline, after cosyntropin 250 µg, and during combined dexamethasone-hydrocortisone treatment. Adrenocorticotropic hormone (ACTH) stimulation produced disproportionate accumulation of adrenal steroid intermediates, which decreased markedly during combined glucocorticoid treatment, supporting ACTH-dependent adrenal steroid accumulation. Targeted POR sequencing and family segregation analysis supported compound heterozygosity for a maternally inherited p.Arg457His allele and an inferred paternal allele carrying p.Arg550Trp and p.Gly413Ser in cis. This case illustrates the value of resolving a long-standing diagnostic suspicion through dynamic steroid profiling, targeted POR sequencing, and segregation analysis; the combined glucocorticoid time point was supportive but not a stand-alone diagnostic test.

PMID 42729616
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PubMedFrontiers in endocrinology2026-09-13

Novel compound heterozygous POR variants in a neonate with Antley-Bixler syndrome and 46,XY DSD: a case report and literature review.

Zhang Wenting W, Zeng Junfeng J, Li Yanjin Y, Xie Cailian C et al.

Cytochrome P450 oxidoreductase deficiency (PORD) is an ultra-rare autosomal recessive disorder within the congenital adrenal hyperplasia (CAH) spectrum, characterized by a broad clinical spectrum involving steroidogenesis defects, genital anomalies, and skeletal abnormalities. We report a phenotypically female neonate with a 46,XY karyotype whose postnatal diagnostic evaluation was initiated after newborn screening revealed elevated 17-hydroxyprogesterone (17-OHP) concentration. The patient presented with mild hypertelorism, mild nasal hypoplasia, and low-set bilateral ears, along with female external genitalia consistent with disorder of sex development (DSD) and anal atresia. Radiological evaluation revealed femoral bowing and subsequent fracture. The craniofacial and skeletal abnormalities were consistent with the features of Antley-Bixler syndrome (ABS). Endocrine evaluation revealed elevated progesterone, markedly reduced testosterone, and secondary hyperaldosteronism. Genetic analysis identified three novel variants in the POR gene (NM_001395413.1): the patient harbored a paternal c.1187_1195dup (p.Pro396_Glu398dup) variant and two maternally inherited variants in cis, c.1447G>A (p.Gly483Ser) and c.1806 + 4_1806 + 28del. Protein structural modeling predicted that the p.Pro396_Glu398dup and p.Gly483Ser may disrupt the flavin adenine dinucleotide (FAD)-binding domain. RNA sequencing (RNA-seq) confirmed that the intronic variant c.1806 + 4_1806 + 28del caused aberrant splicing, resulting in partial intron retention and predicted impairment of the nicotinamide adenine dinucleotide phosphate (NADPH)-binding domain. According to American College of Medical Genetics and Genomics (ACMG) guidelines and incorporating functional evidence, c.1187_1195dup and c.1806 + 4_1806 + 28del were reclassified as likely pathogenic (LP), whereas c.1447G>A remained a variant of uncertain significance (VUS). This study describes a neonate with PORD caused by three novel POR variants and expands the known clinical spectrum of PORD by identifying rare manifestations including anal atresia and hearing loss. RNA-seq provided valuable functional evidence for variant interpretation and facilitated accurate molecular diagnosis. These findings highlight the importance of integrating genetic phasing, transcript-level functional analysis, and comprehensive clinical evaluation for precise diagnosis and counseling in rare endocrine disorders.

PMID 42729307
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PubMedJournal of the National Cancer Institute2026-09-10

Endocrine Biomarker Changes in a Randomised Low-Dose Tamoxifen Trial for Breast Cancer Prevention.

Nash Stephen S, Hammarström Mattias M, Thörngen John-Olof JO, Winqvist Ola O et al.

Tamoxifen reduces breast cancer incidence and recurrence, but uptake for primary prevention remains limited, largely because of concerns regarding adverse effects at the standard 20 mg dose. Understanding systemic endocrine effects of lower tamoxifen doses may help improve future prevention strategies. We analysed data from 1,055 healthy women enrolled in the randomised, double-blind, placebo-controlled KARISMA trial, assigned to placebo or tamoxifen 1, 2.5, 5, 10, or 20 mg daily for 6 months. Plasma concentrations of endocrine biomarkers and tamoxifen metabolites were measured at study end. Associations between randomised tamoxifen dose, circulating metabolite concentrations, and endocrine biomarker plasma concentration (estrogens, androgens, progestogens, cortisol, prolactin, and sex hormone-binding globulin (SHBG)), were evaluated. Tamoxifen dose was associated with measurable endocrine changes after six months, most consistently increased SHBG levels, with additional associations observed for cortisol and hydroxyprogesterone. SHBG demonstrated the clearest dose-response relationship, with increasing levels across tamoxifen dose groups and evidence of attenuated increase at intermediate doses. Large relative differences between placebo and 20 mg tamoxifen were additionally observed for estrone, estrone sulphate and estradiol. Unadjusted analyses of circulating endoxifen showed broadly similar endocrine patterns; however, these associations were substantially attenuated after adjustment for randomised tamoxifen dose. Circulating tamoxifen metabolites were strongly correlated with administered dose and with one another. Low-dose tamoxifen was associated with measurable endocrine changes, particularly in SHBG, cortisol, and hydroxyprogesterone. These findings show endocrine pharmacodynamic responses during low-dose tamoxifen therapy warrants further investigation as a potential component of future individualised prevention and adjuvant endocrine therapy strategies. ClinicalTrials.gov ID: NCT03346200.

PMID 42720596
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PubMedmBio2026-09-10

Sex and age differences in antibody responses to seasonal influenza vaccination are mediated by estrogenic upregulation of NF-κB and TNF signaling in B cells.

Park Han-Sol H-S, Yin Anna A, Zhou Weiqiang W, Wenstedt Eliane F E EFE et al.

Sex differences in the humoral immune responses to the seasonal quadrivalent influenza vaccine (QIV) in young adults (YA; 18-49 years old) or high-dose QIV in old adults (OA; 75+ years old) were analyzed to determine how age-related changes, including in steroids, impact sex differences in B cells. Among YAs, females had greater H3N2, but not H1N1, neutralizing antibody titers, and greater proportions of hemagglutinin (HA)+ CD19+ B cells and HA+ memory B cells than males through 28 days post-vaccination (DPV), that was not observed among OAs. Machine learning algorithms illustrated that baseline (0 DPV) steroids, including 17-hydroxyprogesterone, estrogens, and testosterone, as well as HA+ CD19+ B cells and HA+ antibody-secreting B cells (ASCs), were major predictors of seroconversion at 28 DPV, particularly in YA. Single-cell RNA sequencing demonstrated that CD19+ B cells from YA females had greater transcriptional activity at 7 DPV than YA males, with upregulation of genes with estrogen-response elements (EREs) along NF-κB-mediated TNF signaling pathways in B-cell subsets, which was mitigated in OA. Estradiol treatment of ASCs from YA females, but not males, increased the number and size of HA+ IgG+ cells and expression of ERE genes along the NF-κB-mediated TNF signaling pathway,that was inhibited by an estrogen receptor antagonist. Pharmacological inhibition of either NF-κB or TNF signaling blocked the ability of E2 to upregulate antibody secretion in cells from YA females. This study provides mechanistic insights into estrogen-mediated increases in influenza vaccine-induced antibody responses among reproductive-aged females and suggests a role for estrogen signaling in the reduction of sex differences in vaccine-induced immunity with old age. Sex differences in influenza vaccine-induced immune responses become less pronounced with old age, which we hypothesize could be related to changes in circulating gonadal steroids. Our study shows that after receipt of the seasonal influenza vaccine, young adult females, who have elevated estrogenic activity, have more B cells that recognize influenza hemagglutinin; their B cells have greater activity along estrogen signaling and inflammatory pathways, and mount stronger antibody responses than young adult males, with these sex differences being mitigated in old adults. We identify estrogen as a key driver of sex differences in influenza immunity by showing that ex vivo estradiol increases antibody production by B cells through the estrogen receptor and engagement with NF-κB. These findings help explain the biological basis for sex differences in vaccine immunity and suggest that the hormonal environment, not just chronological age, shapes how well a person responds to vaccination.

PMID 42720319
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PubMedIndian journal of clinical biochemistry : IJCB2026-09-09

Serum 17 Alpha-Hydroxyprogesterone Analysis- Performance Evaluation of Maglumi® X-8 Chemiluminescence Immunoassay.

Ahmed Sibtain S, Siddiqui Ayra A, Lakhani Shan S, Khan Samia S et al.

Congenital Adrenal Hyperplasia (CAH) is primarily caused by 21-hydroxylase deficiency, leading to abnormal adrenal hormone production. Accurate measurement of 17-hydroxyprogesterone (17-OHP) is critical for diagnosing and managing CAH. Traditionally, 17-OHP levels are assessed using ELISA, but methods like chemiluminescent immunoassays (CLIA) offer potential improvements in accuracy and efficiency. We evaluated the Maglumi® X-8 CLIA system for measuring 17-OHP levels and compared its performance with the established ELISA method. The study included serum samples from 39 patients, analyzed for accuracy, precision, linearity, and reportable range. The correlation between CLIA and ELISA results was assessed using regression analysis and Bland-Altman plots. The Maglumi® X-8 CLIA demonstrated acceptable precision, with low coefficients of variation (CVs) at both low and high 17-OHP concentrations. The assay showed strong correlation with the ELISA method (slope: 0.961, intercept: 1.712) and consistent accuracy across a broad reportable range (0.10 to 320 ng/mL). The CLIA system also proved to be faster and more automated, reducing the potential for human error and facilitates faster results for clinical decision making. The Maglumi® X-8 CLIA is a highly precise, accurate, and efficient method for measuring 17-OHP levels, making it a suitable alternative to ELISA in clinical laboratories. Its integration into routine workflows offers faster results and improved reliability.

PMID 42712792
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