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baclofen (Xylka / Baclocur / baclofen, Ethypharm)

✓ Approved

Ethypharm Corp. · GABBR1 · Small Molecule

What is baclofen?

baclofen is a small molecule developed by Ethypharm Corp.. It is approved for therapeutic indications via oral (po).

Drug Profile

Brand NamesXylka, Baclocur, baclofen, Ethypharm
CompanyEthypharm Corp.
Drug ClassSmall Molecule
Molecular TargetGABBR1
RouteOral (PO)
StatusApproved
Sources: ClinicalTrials.gov, Ethypharm Corp.

Mechanism of Action

Molecular Targets

baclofen acts on 1 molecular target:

GABBR1gamma-aminobutyric acid type B receptor subunit 1 (GABABR1, GB1)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

baclofen is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Psychiatric disordersAlcoholism✓ Approved

Related Research Articles

PubMedRinsho shinkeigaku = Clinical neurology2026-05-25

[Beta-propeller protein-associated neurodegeneration (BPAN) associated with heper-CKemia-case report].

Sugiyama Tomoko T, Matsumoto Mizuki M, Okuda Shinya S, Mizobe Marina M et al.

Beta-propeller protein-associated neurodegeneration (BPAN) is a neurodegeneration with brain iron accumulation (NBIA) disorder caused by autophagy abnormalities. Clinical features of BPAN include global developmental delay in early childhood, followed by progression of cognitive dysfunction and parkinsonism in adulthood. A 32-year-old woman diagnosed with BPAN and confirmed by genetic analysis showed motor symptoms that rapidly progressed after the age of 30 years. Baclofen was administered for spasticity, though a high fever and elevated serum CK level were observed, and the symptoms persisted for several months even after stopping the drug. It was suspected that rhabdomyolysis have been due to muscle tissue fragility associated with autophagy impairment in BPAN, in combination with increased muscle tone.

PMID 42178243
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PubMedFrontiers in medicine2026-05-25

Neuromyelitis optica spectrum disorder with painful tonic spasms as the first symptom: a case report.

Zhao Feng F, Wang Yue Y, Gong Jiajun J, Yu Linjie L et al.

Painful tonic spasms (PTS) are an underrecognized initial manifestation of neuromyelitis optica spectrum disorder (NMOSD), presenting before classic neurological deficits in rare cases. A 51-year-old man presented with progressive right lower limb PTS lasting ≤10 s, spreading bilaterally and impairing mobility within 1 month. Neurological examination showed bilateral hyperreflexia and myoclonus. Spinal MRI revealed longitudinally extensive T2 hyperintensity and gadolinium enhancement at T1-T6 levels. Serum aquaporin-4 immunoglobulin G (AQP4-IgG) antibody was positive (1:100), while cerebrospinal fluid AQP4-IgG analysis was negative. Methylprednisolone (0.5 g/day for 3 days, tapered to oral prednisolone) and intravenous immunoglobulin (0.4 g/kg/day for 5 days) significantly reduced spasms. At discharge, he ambulated independently without PTS on prednisolone (60 mg/day), oxcarbazepine (450 mg twice daily), and baclofen (10 mg twice daily). Isolated PTS serves as a potential harbinger of NMOSD, and early diagnosis and precise immunotherapy enables rapid symptom control and attack prevention.

PMID 42180759
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PubMedeNeurologicalSci2026-05-25

Fractures beyond spasms: Progressive encephalomyelitis with rigidity and myoclonus associated with GABA-linked bone fragility.

Shibahara Tomoya T, Kuroda Junya J, Nakane Hiroshi H, Ago Tetsuro T

Progressive encephalomyelitis with rigidity and myoclonus (PERM) is a rare and severe form of stiff-person spectrum disorders, typically associated with painful spasms, brainstem involvement, and autonomic dysfunction. Previous reports have described fractures as an uncommon but recognized complication of PERM, typically attributed to mechanical stress from recurrent spasms; however, disrupted γ- aminobutyric acid (GABA)ergic signaling may also impair bone metabolism. We report a 53-year-old man with seronegative PERM who developed vertebral and pelvic fractures without trauma during hospitalization. Immunotherapy with intravenous immunoglobulin, methylprednisolone, and rituximab improved neurological symptoms, but spasticity persisted. Bone turnover markers remained persistently elevated and bone healing was delayed even 6 months after the fractures. Introduction of intrathecal baclofen resulted in marked reduction of spasticity, normalization of bone markers, improvement in bone mineral density, and radiographic evidence of fracture healing. This case suggests that impaired GABAergic signaling in PERM may contribute to high-turnover bone metabolism and skeletal fragility, in addition to mechanical stress. Awareness of this potential complication is important, as early initiation of immunotherapy combined with GABA-enhancing therapy may improve both neurological and skeletal outcomes.

PMID 42180596
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PubMedJournal of pediatric gastroenterology and nutrition2026-05-18

Rumination syndrome and eosinophilic esophagitis in children: Defining the relationship.

Yang Dennis D, Lu Peter L PL, Khoo Janice S JS, Sanchez Raul E RE et al.

While rumination syndrome (RS) and eosinophilic esophagitis (EoE) can share overlapping symptoms, their association remains unclear. Our objective was to explore the relationship between RS and EoE and understand the clinical course of children with both conditions. We conducted a retrospective cohort study of children with RS and EoE evaluated at our institution from 2016 to 2023. Patient characteristics, symptoms, treatments, and outcomes were compared. Among 230 children with RS, 22 (10%) had both RS and EoE (77% male, median age 18 years). Sixteen (70%) were first diagnosed with EoE, two (9%) with RS, and four (18%) concurrently. Most (91%) presented with regurgitation; 64% reported dysphagia at some point. EoE treatment led to mucosal remission in 73% and improved dysphagia in 41%, but 86% continued to experience regurgitation. RS treatment, including behavioral therapy and baclofen, improved or resolved regurgitation in 64%. Peak eosinophil counts were similar in children with and without dysphagia (43 vs. 51 eos/HPF, p = 0.589). Those without dysphagia responded better to RS treatment (67% vs. 22%), though the difference was not statistically significant. Among those diagnosed with EoE first, RS diagnosis occurred after a median of 15.4 months. EoE was more prevalent among children with RS than in the general population. Regurgitation seemed more responsive to RS treatment than EoE treatment. In patients with EoE and persistent regurgitation, RS should be considered, especially when symptoms persist despite EoE remission.

PMID 42148519
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PubMedCureus2026-05-15

Baclofen in Refractory Dyspepsia With Chronic Belching: A Case Report.

Rocha Mariana M, Silva Lisete L, Coelho Francisco F

Dyspeptic symptoms associated with chronic belching may substantially affect quality of life and can be challenging to manage in clinical practice. We report the case of a 40-year-old nulligravid woman with depressive disorder, bilateral cochlear implants due to deafness, a hiatal hernia, and a previous pulmonary embolism. Her medications included desvenlafaxine, multiple benzodiazepines, mirtazapine, trazodone, and apixaban. She presented with an 18-month history of persistent belching, heartburn, early satiety, and intermittent dry cough, severely impairing her quality of life and leading to social isolation. Additionally, her symptoms intensified during anxiety and depressive episodes. Physical examination was unremarkable, and upper gastrointestinal endoscopy revealed only a small hiatal hernia and mild gastropathy, with no evidence of Helicobacter pylori infection. Despite repeated medical evaluations and multiple therapeutic approaches, including proton pump inhibitors, sucralfate, domperidone, simethicone, and bilastine, symptoms persisted without significant improvement. Functional dyspepsia was therefore suspected, and treatment with baclofen 10 mg twice daily was initiated, resulting in significant clinical improvement and enhanced quality of life. This report highlights the potential benefit of baclofen in patients with persistent belching and dyspeptic symptoms refractory to conventional therapy, particularly in the context of psychiatric comorbidity.

PMID 42137706
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PubMedBMJ case reports2026-05-15

Intrathecal baclofen pump failure due to pain-induced autonomic dysreflexia.

Khawer Ahmed A, Jain Ankur A, Naji Luay L, Sheikh Amaan A et al.

Intrathecal baclofen (ITB) therapy is an established treatment for severe refractory spasticity. Although a positive ITB trial confirms efficacy, long-term success can sometimes be limited by mechanical or hardware complications. We describe a patient with multiple sclerosis (MS) and spastic quadriplegia who underwent ITB pump implantation in the posterolateral flank region, a non-standard site selected due to anatomical limitations. The pump provided marked spasticity relief, allowing discontinuation of oral antispasmodics. However, within weeks, the patient developed persistent pump-site pain and recurrent autonomic dysreflexia (AD), an uncommon manifestation in MS that was resistant to treatment. Infectious and other mechanical causes were excluded, and clinical assessment determined that pain from the atypical pump placement was the likely precipitant. The pump was explanted 1 month later, resolving the patient's AD but resulting in recurrence of severe spasticity. This case underscores the need for individualised pump planning to prevent hardware-related complications.

PMID 42134868
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