RARE DISEASE
Infantile spasms syndrome
Infantile spasms syndrome
Infantile spasms syndrome
Synonyms: West syndrome
Synonyms: West syndrome
Synonyms: West syndrome
Drug discovery
18
drugs
With orphan designations
Overview
Infantile Spasms Syndrome (ISS), formerly West syndrome, is a severe developmental and epileptic encephalopathy characterized by epileptic spasms (tonic-flexor/extensor clusters), hypsarrhythmia on EEG, and neurodevelopmental regression. Onset typically occurs between 3-12 months, with peak incidence at 4-7 months [1][4][12]. Etiologies include structural brain abnormalities (e.g., hypoxic injury, tuberous sclerosis), genetic mutations, and metabolic disorders, though 20-30% remain cryptogenic [4][11][17]. Early treatment with hormonal therapy or vigabatrin improves developmental outcomes, but delayed diagnosis remains common due to subtle seizure semiology [3][5][16].
Burden
Mortality: 3–33%, often linked to underlying etiology or sepsis [4][14].
Neurodevelopmental: 70–90% experience cognitive impairment; 30–50% progress to refractory epilepsy (e.g., Lennox-Gastaut syndrome) [4][5][17].
Economic: High costs from therapies, hospitalizations, and caregiver work loss (median 12 days/year) [9][16].
Therapies
First-line: Hormonal therapy (ACTH/prednisolone) or vigabatrin (preferred for tuberous sclerosis) [3][5][13].
Second-line: Ketogenic diet, epilepsy surgery (for focal lesions), or adjunctive antiseizure medications (e.g., topiramate) [5][16].
Treatment response monitored by clinical spasms cessation and EEG normalization within 2 weeks [3][8].
Categories: rare genetic diseases, rare neurological diseases
Research Papers
1,122 drug discovery papers about Infantile spasms syndrome, with 2 first-in-class and 16 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,122 drug discovery papers about Infantile spasms syndrome, with 2 first-in-class and 16 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-10 | Electrophysiological readout of ACTH-related neuroendocrine–immunomodulatory treatment response in infantile epileptic spasms syndrome: suppression of scalp ripple propagation networks
Background Adrenocorticotropic hormone (ACTH) is a first-line neuroendocrine–immunomodulatory therapy for infantile epileptic spasms syndrome (IESS), but noninvasive electrophysiological markers that objectively reflect treatment-related network changes remain limited. We investigated whether scalp putative ripple propagation network metrics provide markers of adrenocorticotropic hormone-related network response and early relapse risk. Methods In this retrospective multicenter study, children receiving first-time ACTH therapy were included if paired pre- and post-treatment scalp electroencephalography (EEG) recordings and at least 6 months of follow-up were available. Five-minute artifact-free interictal slow-wave-sleep segments were analyzed. Putative ripple propagation networks were constructed using a time-delay-based method. The primary metrics were post-treatment total network connection strength (total NCS) and its reduction ratio, with ripple count and ripple-count reduction ratio used as rate-based comparators. Results Sixty-one children were included; 27 achieved acute spasm freedom, and seven of these relapsed within 6 months. Baseline ripple count and network metrics did not distinguish subsequent responders from nonresponders. After adrenocorticotropic hormone therapy, acute responders showed marked suppression of putative ripple propagation network metrics, whereas nonresponders showed reduction in ripple count without comparable reductions in network metrics. Post-treatment total network connection strength and its reduction ratio differentiated acute responders from nonresponders, with areas under the curve (AUC) of 0.902 and 0.899, respectively. In exploratory analyses among acute responders, greater total network connection strength reduction was associated with sustained 6-month spasm freedom, although relapse-related estimates were limited by the small number of events. Conclusion Scalp putative ripple propagation networks provide a noninvasive electrophysiological readout of ACTH-related network modulation in infantile epileptic spasms syndrome. In contrast to ripple count alone, network connection strength captured whether treatment was accompanied by disruption of propagating high-frequency activity. These findings suggest that total NCS and its reduction ratio may serve as candidate markers of adrenocorticotropic hormone-related neuroendocrine–immunomodulatory therapy response, while their relevance to relapse risk requires prospective validation.
2026-07-21 | Vigabatrin therapy for infantile epileptic spasms syndrome with periventricular leukomalacia.
Infantile epileptic spasm syndrome (IESS) and periventricular leukomalacia (PVL) cause developmental regression and poor neurological outcomes. The primary treatments for IESS include adrenocorticotropin and vigabatrin; however, no study has yet evaluated the efficacy of vigabatrin for patients with both IESS and PVL. Therefore, we investigated the efficacy and safety of vigabatrin in patients with both disorders. We retrospectively enrolled patients with IESS and PVL who were treated with vigabatrin. We performed ophthalmological examinations and electroretinography (ERG) before and after vigabatrin. Treatment response was defined as complete cessation of epileptic spasms and no other seizures after 3 months of vigabatrin. We investigated the responses to vigabatrin, doses used, and adverse effects. Seven patients (5 males) with IESS and PVL were treated with vigabatrin. The median age at vigabatrin initiation was 10 (range, 7-25) months. Three of the seven patients (42.8%) showed complete clinical cessation of epileptic spasms after 3 months of vigabatrin. The median duration between vigabatrin initiation and epileptic spasm cessation was 5 (range: 3-61) days. The adverse effects included poor feeding (n = 1) and abnormal ERG findings during vigabatrin therapy (n = 2). These ERG findings normalized after vigabatrin was reduced or stopped. No respiratory failure, increased secretion, or somnolence was observed. Vigabatrin was effective for treating 42.8% of our patients with both IESS and PVL. Vigabatrin was well tolerated, with only a mild effect observed in patients with IESS and PVL. Some treatment-related ERG abnormalities were normalized after vigabatrin was reduced or stopped.
2026-06-26 | HMGB1-TLR4 signaling-mediated neuroinflammation contributes to the pathogenesis of infantile epileptic spasms syndrome in rats
Background: Infantile epileptic spasm syndrome (IESS) is a severe age-dependent epileptic encephalopathy in infancy with poor prognosis and unclear pathogenesis. Neuroinflammation plays a pivotal role in epileptogenesis, and the high-mobility group box 1 protein (HMGB1)-Toll-like receptor 4 (TLR4) axis acts as a core mediator of neuroinflammation. However, its specific role in IESS remains elusive. Objective: This study aimed to explore the HMGB1-TLR4-mediated neuroinflammatory mechanism in a rat model of IESS induced by prenatal stress combined with NMDA, and to evaluate the effects of anti-HMGB1 neutralizing antibody and adrenocorticotropic hormone (ACTH) on epileptic seizures and neuroinflammation, so as to provide novel therapeutic targets for clinical practice. Methods: Pregnant Sprague-Dawley rats were randomly divided into prenatal stress (PS) and non-prenatal stress (NPS) groups. PS rats received cold water immersion and hot air drying, while NPS rats were reared normally. On postnatal day 12 (P12), offspring in the PS group were intraperitoneally injected with NMDA to establish the IESS model, and the NPS group was assigned to blank control (BC) and negative control (NC) subgroups. Model rats were randomly divided into ACTH, anti-HMGB1, ACTH+anti-HMGB1, normal saline, and untreated groups. After intervention on P13, NMDA was re-administered, and seizure latency and severity score were recorded. At the end of the experiment, the expression of HMGB1 and TLR4 in brain tissue was detected, HMGB1 co-localization was observed, and the levels of iNOS, Arg1 and cytokines (IL-1β, IL-2R, IL-8, TNF-α) were measured. Results: Prenatal stress combined with NMDA successfully established a stable IESS model in young rats. The expression of HMGB1, TLR4, iNOS, IL-1β, IL-2R, IL-8 and TNF-α was significantly upregulated, while Arg1 was markedly downregulated. Treatment with ACTH, anti-HMGB1, and their combination prolonged seizure latency, reduced seizure severity, downregulated HMGB1 and TLR4 expression, suppressed HMGB1 levels in neurons, astrocytes and activated microglia, inhibited iNOS and proinflammatory cytokines, and promoted Arg1 expression, with the combined intervention showing the optimal efficacy. Conclusion: Prenatal stress combined with NMDA activates the HMGB1/TLR4 pathway and neuroinflammation in IESS rats. ACTH and anti-HMGB1, alone or in combination, alleviate neuroinflammation by inhibiting this pathway to ameliorate IESS, and the combined therapy yields the best therapeutic effect.
2026-06-09 | PARS2 deficiency impairs mitochondrial homeostasis and activates ferroptotic to drive developmental and epileptic encephalopathy.
PARS2, encodes a mitochondrial aminoacyl-tRNA synthetase associated with developmental and epileptic encephalopathy (DEE), a severe neurological disorder characterized by refractory epilepsy and intellectual disability. While genetic associations between PARS2 and DEE have been established, the underlying molecular mechanisms remain poorly understood. This study integrates genetic analyses of clinical cases of infantile epileptic spasms syndrome (IESS) with functional assessments in PARS2-deficient animal models and cell models to elucidate these mechanisms. Our findings indicate that PARS2 deficiency disrupts mitochondrial integrity and impairs oxidative phosphorylation, resulting in elevated intracellular calcium levels. This calcium overload activates CaMKK2-AMPK-Drp1 signaling, promoting excessive mitochondrial fission and PINK1-Parkin-mediated mitophagy, ultimately leading to degradation of GPX4 and subsequent ferroptosis. Notably, pharmacological inhibition of Drp1 using Mdivi-1 successfully rescued mitochondrial fragmentation and mitigated ferroptosis. These results unveil a novel calcium-mitophagy-ferroptosis pathway as a crucial mechanism in PARS2-related DEE and propose a potential therapeutic strategy for DEE.
2026-05-20 | Blockade of Corticotropin-Releasing Hormone Receptor 1 Receptors in the Arcuate Nucleus May Effectively Treat Infantile Epileptic Spasms Syndrome
Targeting Corticotropin-Releasing Hormone Receptor 1 Signaling in Experimental Infantile Epileptic Spasms Syndrome: Evidence for Route-Dependent Efficacy. Chachua T, Yum MS, Chern CR, Vieira K, Velíšková J, Velíšek L. Targeting CRHR1 Signaling in Experimental Infantile Epileptic Spasms Syndrome: Evidence for Route-Dependent Efficacy. Children (Basel). 2026 Jan 14;13(1):125. doi: 10.3390/children13010125. PMID: 41597133; PMCID: PMC12840083. Background/Objectives: Infantile epileptic spasms syndrome (IESS) is a severe epilepsy of infancy. Corticotropin (ACTH) and vigabatrin are the only FDA-approved therapies. The efficacy of ACTH together with the strong convulsant effects of corticotropin-releasing hormone (CRH) suggests that excess CRH, secondary to impaired ACTH feedback, may contribute to spasms. We therefore hypothesized that CRH receptor 1 (CRHR1) antagonists would suppress spasms in a route- and drug-dependent manner. Methods: Using our validated rat model of IESS, in which prenatal priming with betamethasone was followed by postnatal triggering of spasms with N-methyl-D-aspartic acid (NMDA), we tested two CRHR1 antagonists, CP376395 and SN003, delivered intracranially (via intracerebroventricular or intraparenchymal infusion) or systemically. Results: Intracerebroventricular infusion of both antagonists suppressed spasms, with CP376395 providing more consistent effects. Intraparenchymal administration into the hypothalamic arcuate nucleus also reduced spasms, whereas misses into the mammillary bodies were ineffective, highlighting site specificity. Systemic administration yielded divergent results: SN003 robustly suppressed spasms, whereas CP376395 unexpectedly exacerbated them. No sex differences were observed. Conclusions: These findings demonstrate that CRHR1 blockade modifies experimental spasms in a route- and drug-specific manner and implicates discrete hypothalamic circuits, particularly those including the arcuate nucleus, in spasm generation. The divergent systemic responses between CP376395 and SN003 likely reflect differences in CRHR1 engagement (competitive and non-competitive antagonism, respectively) as well as differences in binding properties that may include differential network interactions beyond local CRH signaling or duration of receptor occupancy. In conclusion, SN003 may be a better option than CP376395 for further development as a CRHR1-targeted therapy pending additional pharmacokinetic/pharmacodynamic studies. Further work should explore dosing paradigms of CP376395 to determine if a therapeutic range for CP376395 exists.
2026-08-10 | Electrophysiological readout of ACTH-related neuroendocrine–immunomodulatory treatment response in infantile epileptic spasms syndrome: suppression of scalp ripple propagation networks
Background Adrenocorticotropic hormone (ACTH) is a first-line neuroendocrine–immunomodulatory therapy for infantile epileptic spasms syndrome (IESS), but noninvasive electrophysiological markers that objectively reflect treatment-related network changes remain limited. We investigated whether scalp putative ripple propagation network metrics provide markers of adrenocorticotropic hormone-related network response and early relapse risk. Methods In this retrospective multicenter study, children receiving first-time ACTH therapy were included if paired pre- and post-treatment scalp electroencephalography (EEG) recordings and at least 6 months of follow-up were available. Five-minute artifact-free interictal slow-wave-sleep segments were analyzed. Putative ripple propagation networks were constructed using a time-delay-based method. The primary metrics were post-treatment total network connection strength (total NCS) and its reduction ratio, with ripple count and ripple-count reduction ratio used as rate-based comparators. Results Sixty-one children were included; 27 achieved acute spasm freedom, and seven of these relapsed within 6 months. Baseline ripple count and network metrics did not distinguish subsequent responders from nonresponders. After adrenocorticotropic hormone therapy, acute responders showed marked suppression of putative ripple propagation network metrics, whereas nonresponders showed reduction in ripple count without comparable reductions in network metrics. Post-treatment total network connection strength and its reduction ratio differentiated acute responders from nonresponders, with areas under the curve (AUC) of 0.902 and 0.899, respectively. In exploratory analyses among acute responders, greater total network connection strength reduction was associated with sustained 6-month spasm freedom, although relapse-related estimates were limited by the small number of events. Conclusion Scalp putative ripple propagation networks provide a noninvasive electrophysiological readout of ACTH-related network modulation in infantile epileptic spasms syndrome. In contrast to ripple count alone, network connection strength captured whether treatment was accompanied by disruption of propagating high-frequency activity. These findings suggest that total NCS and its reduction ratio may serve as candidate markers of adrenocorticotropic hormone-related neuroendocrine–immunomodulatory therapy response, while their relevance to relapse risk requires prospective validation.
2026-07-21 | Vigabatrin therapy for infantile epileptic spasms syndrome with periventricular leukomalacia.
Infantile epileptic spasm syndrome (IESS) and periventricular leukomalacia (PVL) cause developmental regression and poor neurological outcomes. The primary treatments for IESS include adrenocorticotropin and vigabatrin; however, no study has yet evaluated the efficacy of vigabatrin for patients with both IESS and PVL. Therefore, we investigated the efficacy and safety of vigabatrin in patients with both disorders. We retrospectively enrolled patients with IESS and PVL who were treated with vigabatrin. We performed ophthalmological examinations and electroretinography (ERG) before and after vigabatrin. Treatment response was defined as complete cessation of epileptic spasms and no other seizures after 3 months of vigabatrin. We investigated the responses to vigabatrin, doses used, and adverse effects. Seven patients (5 males) with IESS and PVL were treated with vigabatrin. The median age at vigabatrin initiation was 10 (range, 7-25) months. Three of the seven patients (42.8%) showed complete clinical cessation of epileptic spasms after 3 months of vigabatrin. The median duration between vigabatrin initiation and epileptic spasm cessation was 5 (range: 3-61) days. The adverse effects included poor feeding (n = 1) and abnormal ERG findings during vigabatrin therapy (n = 2). These ERG findings normalized after vigabatrin was reduced or stopped. No respiratory failure, increased secretion, or somnolence was observed. Vigabatrin was effective for treating 42.8% of our patients with both IESS and PVL. Vigabatrin was well tolerated, with only a mild effect observed in patients with IESS and PVL. Some treatment-related ERG abnormalities were normalized after vigabatrin was reduced or stopped.
2026-06-26 | HMGB1-TLR4 signaling-mediated neuroinflammation contributes to the pathogenesis of infantile epileptic spasms syndrome in rats
Background: Infantile epileptic spasm syndrome (IESS) is a severe age-dependent epileptic encephalopathy in infancy with poor prognosis and unclear pathogenesis. Neuroinflammation plays a pivotal role in epileptogenesis, and the high-mobility group box 1 protein (HMGB1)-Toll-like receptor 4 (TLR4) axis acts as a core mediator of neuroinflammation. However, its specific role in IESS remains elusive. Objective: This study aimed to explore the HMGB1-TLR4-mediated neuroinflammatory mechanism in a rat model of IESS induced by prenatal stress combined with NMDA, and to evaluate the effects of anti-HMGB1 neutralizing antibody and adrenocorticotropic hormone (ACTH) on epileptic seizures and neuroinflammation, so as to provide novel therapeutic targets for clinical practice. Methods: Pregnant Sprague-Dawley rats were randomly divided into prenatal stress (PS) and non-prenatal stress (NPS) groups. PS rats received cold water immersion and hot air drying, while NPS rats were reared normally. On postnatal day 12 (P12), offspring in the PS group were intraperitoneally injected with NMDA to establish the IESS model, and the NPS group was assigned to blank control (BC) and negative control (NC) subgroups. Model rats were randomly divided into ACTH, anti-HMGB1, ACTH+anti-HMGB1, normal saline, and untreated groups. After intervention on P13, NMDA was re-administered, and seizure latency and severity score were recorded. At the end of the experiment, the expression of HMGB1 and TLR4 in brain tissue was detected, HMGB1 co-localization was observed, and the levels of iNOS, Arg1 and cytokines (IL-1β, IL-2R, IL-8, TNF-α) were measured. Results: Prenatal stress combined with NMDA successfully established a stable IESS model in young rats. The expression of HMGB1, TLR4, iNOS, IL-1β, IL-2R, IL-8 and TNF-α was significantly upregulated, while Arg1 was markedly downregulated. Treatment with ACTH, anti-HMGB1, and their combination prolonged seizure latency, reduced seizure severity, downregulated HMGB1 and TLR4 expression, suppressed HMGB1 levels in neurons, astrocytes and activated microglia, inhibited iNOS and proinflammatory cytokines, and promoted Arg1 expression, with the combined intervention showing the optimal efficacy. Conclusion: Prenatal stress combined with NMDA activates the HMGB1/TLR4 pathway and neuroinflammation in IESS rats. ACTH and anti-HMGB1, alone or in combination, alleviate neuroinflammation by inhibiting this pathway to ameliorate IESS, and the combined therapy yields the best therapeutic effect.
2026-06-09 | PARS2 deficiency impairs mitochondrial homeostasis and activates ferroptotic to drive developmental and epileptic encephalopathy.
PARS2, encodes a mitochondrial aminoacyl-tRNA synthetase associated with developmental and epileptic encephalopathy (DEE), a severe neurological disorder characterized by refractory epilepsy and intellectual disability. While genetic associations between PARS2 and DEE have been established, the underlying molecular mechanisms remain poorly understood. This study integrates genetic analyses of clinical cases of infantile epileptic spasms syndrome (IESS) with functional assessments in PARS2-deficient animal models and cell models to elucidate these mechanisms. Our findings indicate that PARS2 deficiency disrupts mitochondrial integrity and impairs oxidative phosphorylation, resulting in elevated intracellular calcium levels. This calcium overload activates CaMKK2-AMPK-Drp1 signaling, promoting excessive mitochondrial fission and PINK1-Parkin-mediated mitophagy, ultimately leading to degradation of GPX4 and subsequent ferroptosis. Notably, pharmacological inhibition of Drp1 using Mdivi-1 successfully rescued mitochondrial fragmentation and mitigated ferroptosis. These results unveil a novel calcium-mitophagy-ferroptosis pathway as a crucial mechanism in PARS2-related DEE and propose a potential therapeutic strategy for DEE.
2026-05-20 | Blockade of Corticotropin-Releasing Hormone Receptor 1 Receptors in the Arcuate Nucleus May Effectively Treat Infantile Epileptic Spasms Syndrome
Targeting Corticotropin-Releasing Hormone Receptor 1 Signaling in Experimental Infantile Epileptic Spasms Syndrome: Evidence for Route-Dependent Efficacy. Chachua T, Yum MS, Chern CR, Vieira K, Velíšková J, Velíšek L. Targeting CRHR1 Signaling in Experimental Infantile Epileptic Spasms Syndrome: Evidence for Route-Dependent Efficacy. Children (Basel). 2026 Jan 14;13(1):125. doi: 10.3390/children13010125. PMID: 41597133; PMCID: PMC12840083. Background/Objectives: Infantile epileptic spasms syndrome (IESS) is a severe epilepsy of infancy. Corticotropin (ACTH) and vigabatrin are the only FDA-approved therapies. The efficacy of ACTH together with the strong convulsant effects of corticotropin-releasing hormone (CRH) suggests that excess CRH, secondary to impaired ACTH feedback, may contribute to spasms. We therefore hypothesized that CRH receptor 1 (CRHR1) antagonists would suppress spasms in a route- and drug-dependent manner. Methods: Using our validated rat model of IESS, in which prenatal priming with betamethasone was followed by postnatal triggering of spasms with N-methyl-D-aspartic acid (NMDA), we tested two CRHR1 antagonists, CP376395 and SN003, delivered intracranially (via intracerebroventricular or intraparenchymal infusion) or systemically. Results: Intracerebroventricular infusion of both antagonists suppressed spasms, with CP376395 providing more consistent effects. Intraparenchymal administration into the hypothalamic arcuate nucleus also reduced spasms, whereas misses into the mammillary bodies were ineffective, highlighting site specificity. Systemic administration yielded divergent results: SN003 robustly suppressed spasms, whereas CP376395 unexpectedly exacerbated them. No sex differences were observed. Conclusions: These findings demonstrate that CRHR1 blockade modifies experimental spasms in a route- and drug-specific manner and implicates discrete hypothalamic circuits, particularly those including the arcuate nucleus, in spasm generation. The divergent systemic responses between CP376395 and SN003 likely reflect differences in CRHR1 engagement (competitive and non-competitive antagonism, respectively) as well as differences in binding properties that may include differential network interactions beyond local CRH signaling or duration of receptor occupancy. In conclusion, SN003 may be a better option than CP376395 for further development as a CRHR1-targeted therapy pending additional pharmacokinetic/pharmacodynamic studies. Further work should explore dosing paradigms of CP376395 to determine if a therapeutic range for CP376395 exists.
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Drug Discovery Landscape
18 orphan drug designations for Infantile spasms syndrome, including 3 approved therapies.
18 orphan drug designations for Infantile spasms syndrome, including 3 approved therapies.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Tricaprilin | small molecules | EMA | 2022-11-10 | — | Veristat Spain S.L. |
vigabatrin [Vigafyde] | small molecules | FDA | 2022-10-12 | 2024-06-17 | Pyros Pharmaceuticals, Inc. |
tricaprilin | small molecules | FDA | 2020-10-27 | — | Cerecin, Inc. |
Synthetic-adrenocorticotropic hormone | peptides | FDA | 2020-09-25 | — | Amzell B.V. |
vigabatrin and cosyntropin | small molecules | FDA | 2017-11-01 | — | West Therapeutic Development, LLC |
Cannabidiol | small molecules | EMA | 2017-10-16 | — | Jazz Pharmaceuticals Ireland Limited |
cosyntropin | peptides | FDA | 2017-08-02 | — | West Therapeutic Development, LLC |
1-(2-chlorophenyl)-1-(S)-hydroxy-2-(S)-carbamoyloxy-propane | small molecules | FDA | 2017-03-20 | — | Bio-Pharm Solutions, Co., Ltd. |
tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride | small molecules | FDA | 2016-06-20 | — | Anavex Life Sciences Corp. |
cannabidiol | small molecules | FDA | 2016-06-13 | — | Jazz Pharmaceuticals Research UK Limited |
cannabidiol | small molecules | FDA | 2015-07-23 | — | Benuvia Operations LLC |
tetracosactide hexaacetate (beta 1-24-corticotrophin) | peptides | FDA | 2012-10-31 | — | Cerium Pharmaceuticals, Inc. |
carisbamate | small molecules | FDA | 2012-03-16 | — | SK Life Science, Inc. |
(1s,3s)-3-amino-4-(difluoromethylene)cyclopentanecarboxylic acid hydrochloride [CPP-115] | small molecules | EMA | 2012-02-09 | — | Catalent Pharma Solutions Limited |
(1S,3S)-3-amino-4-(difluoromethylene)cyclopentanecarboxylic acid hydrochloride, (1S,3S)-3-amino-4-difluoromethylenyl-1-cyclopentanoic acid hydrochloride | small molecules | FDA | 2010-09-15 | — | Catalyst Pharmaceuticals, Inc. |
repository corticotropin or adrenocorticotropic hormone [H.P. Acthar Gel] | peptides | FDA | 2003-05-21 | 2010-10-15 | Questcor Pharmaceuticals, Inc. |
vigabatrin [Sabril] | small molecules | FDA | 2000-06-12 | 2009-08-21 | H. Lundbeck A/S |
Ganaxolone | small molecules | FDA | 1994-05-25 | — | Immedica Pharma AB |
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