AI Drug Discovery for Pharma and Biotech

Drug discovery

7

drugs

With orphan designations

Overview

Epilepsy is a chronic neurological disorder characterized by recurrent, unprovoked seizures due to abnormal neuronal activity. It affects all ages and has diverse etiologies, including genetic, structural, and unknown causes. While ~70% achieve seizure control with antiseizure medications (ASMs), 30% develop drug-resistant epilepsy, requiring advanced therapies. The condition carries significant mortality risks, including SUDEP (sudden unexpected death in epilepsy) [4][6][16].

Population

  • Global: Affects ~51.7 million people (24.2 million idiopathic; 27.5 million secondary) [9][19], with age-standardized prevalence of 307.38/100,000 [2].

  • Demographics: Highest incidence in children (0–14 years: 61/100,000) [2][19]; mortality peaks in adults ≥70 years (5.67/100,000) [2][19].

  • Disparities: 80% of cases occur in low- and middle-income countries, with treatment gaps exceeding 75% in some regions [16][19].

Burden

  • Mortality: Age-standardized death rate of 1.74/100,000 globally [2][19]; SUDEP accounts for 1/1,000 annual deaths in epilepsy patients [4][6].

  • Disability: 177.85 DALYs/100,000 globally, with males disproportionately affected (201.29 vs. 154.25 in females) [2][19].

  • Comorbidities: 20–30% have depression/anxiety; 27-fold higher sudden death risk vs. general population [6][16].

Therapies

  • First-line: ASMs (levetiracetam, valproate) control seizures in ~67% of patients [1][8][16].

  • Advanced options: Surgical resection, neurostimulation (vagus nerve stimulation, responsive neurostimulation), and ketogenic diet for drug-resistant cases [3][8][13].

  • Emerging therapies: Cenobamate, cannabidiol, and non-invasive neuromodulation (e.g., transcranial magnetic stimulation) [3][13][18].

Categories: rare genetic diseases, rare neurological diseases

Research Papers

3,259 drug discovery papers about Epilepsy syndrome, with 2 first-in-class and 10 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

3,259 drug discovery papers about Epilepsy syndrome, with 2 first-in-class and 10 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-10 | Magnetic resonance-guided laser interstitial thermal therapy for mesial temporal lobe epilepsy (MINE): a single-centre, prospective, randomised, open-label, blinded-endpoint controlled trial protocol in China.

Drug-resistant mesial temporal lobe epilepsy (mTLE) is a common and disabling focal epilepsy syndrome for which surgery offers the best chance of seizure freedom. Traditional open surgery (eg, anterior temporal lobectomy or selective amygdalohippocampectomy) is effective but may be associated with perioperative morbidity and neurocognitive sequelae. Magnetic resonance-guided laser interstitial thermal therapy (MRgLITT) is a minimally invasive alternative with encouraging observational outcomes but high-quality comparative evidence versus open surgery remains limited. The MINE trial is designed to compare MRgLITT with open surgery in patients with mTLE. MINE is a prospective, randomised, open-label, blinded-endpoint trial. Eligible adults with drug-resistant TLE who complete a comprehensive presurgical evaluation and are deemed suitable for surgical treatment will be enrolled at participating centres. Participants will be randomised 1:1 (block randomisation) to receive MRgLITT or open surgery. Because of the nature of surgical interventions, participants and treating teams will not be blinded; however, outcome assessors and statistical analysts will remain blinded to treatment allocation. The primary outcome is seizure-free rate at 12 months after surgery, defined as International League Against Epilepsy (ILAE) Class 1. Secondary outcomes include: (1) broader seizure outcomes (ILAE class distribution, seizure frequency and seizure severity) assessed at 6 and 12 months; (2) cognitive and memory function assessed at 6 and 12 months; (3) postoperative safety outcomes collected from surgery through discharge and at follow-up visits at 6 and 12 months, and (4) healthcare utilisation and health-economic outcomes: length of hospital stay measured during hospitalisation; and reoperation, antiseizure medication reduction and quality of life assessed at 6 and 12 months. Safety outcomes include perioperative adverse events (AEs) and serious AEs (SAEs), collected from surgery through discharge and at follow-up visits at 6 and 12 months. This trial is powered as a non-inferiority study on the primary outcome, which based on prior data reporting 12-month seizure-free rates of 76.9% for MRgLITT and 76.0% for open surgery, the assumed absolute difference is 0.009. The non-inferiority margin was set at -0.23, informed by the minimum acceptable benefit threshold reported in patient preference research. The study has received ethics approval from the Institutional Review Board of Beijing Tiantan Hospital (KY2025-073-01). Written informed consent will be obtained from all participants prior to enrolment. Results will be disseminated through peer-reviewed publications and academic presentations. NCT06720922.

Open article ↗



2026-07-05 | Cenobamate use in super-refractory status epilepticus: A report of three cases.

Super-refractory status epilepticus (SRSE) is a neurological emergency with high morbidity and mortality. Cenobamate, a novel antiseizure medication, may be helpful in managing SRSE, but evidence is limited. This retrospective case series reports the use of cenobamate as add-on therapy in the management of three cases of SRSE. Median age at status epilepticus (SE) onset was 28 years (range 27-75). The etiologies of SRSE included one case of febrile infection-related epilepsy syndrome (FIRES), one patient with a probable genetic etiology (SCN7A variant of uncertain significance), and one case of unknown etiology. Cenobamate was introduced at a median of 27 days (range 13-103) after SE onset. Resolution of SRSE was observed after a median of 7 days (range 3-30) once cenobamate was started. Median dose of cenobamate at SRSE cessation was 25 mg/day (range 12.5-50 mg/day), and the median maintenance dose at the time of discharge was 200 mg/day (range 100-400 mg/day). Two patients died and one patient achieved functional independence. No severe medication side effects, specifically drug reaction with eosinophilia and systemic symptoms (DRESS), were observed. Cenobamate may have a role in the management of SRSE. Further studies are needed to define the optimal timing of initiation, titration strategy, and target dose of cenobamate in the context of SE.

Open article ↗



2026-06-29 | Anakinra for tocilizumab-refractory febrile infection-related epilepsy syndrome with normal IL-1β levels: a case report.

Febrile infection-related epilepsy syndrome (FIRES) is a severe epileptic encephalopathy with limited treatment options. Neuroinflammation, particularly involving IL-1β and IL-6, is a key driver. Evidence on sequential use of IL-6 receptor blockade (tocilizumab) followed by IL-1 receptor blockade (anakinra) is scarce, especially when IL-1β levels are normal. A previously healthy 6-year-old boy developed super-refractory status epilepticus six days after influenza A infection. He failed multiple antiseizure medications, anesthetics, corticosteroids, intravenous immunoglobulin, ketogenic diet, and mild therapeutic hypothermia. Cerebrospinal fluid (CSF) and serum cytokine testing showed elevated IL-6 but normal IL-1β. After initial improvement with tocilizumab, seizures relapsed. Tocilizumab retreatment and plasma exchange were ineffective. Switching to daily subcutaneous anakinra led to rapid seizure control within five days. He was weaned off anesthetics, extubated, and eventually discharged with mild cognitive dysfunction (PCPC score 2). At one-month follow-up, he had rare brief seizures and attended school. This case demonstrates that anakinra can be effective in tocilizumab-refractory FIRES even when CSF and serum IL-1β levels are normal. Anakinra should be considered as a second-line option in selected patients, regardless of IL-1β biomarker status.

Open article ↗



2026-07-10 | Magnetic resonance-guided laser interstitial thermal therapy for mesial temporal lobe epilepsy (MINE): a single-centre, prospective, randomised, open-label, blinded-endpoint controlled trial protocol in China.

Drug-resistant mesial temporal lobe epilepsy (mTLE) is a common and disabling focal epilepsy syndrome for which surgery offers the best chance of seizure freedom. Traditional open surgery (eg, anterior temporal lobectomy or selective amygdalohippocampectomy) is effective but may be associated with perioperative morbidity and neurocognitive sequelae. Magnetic resonance-guided laser interstitial thermal therapy (MRgLITT) is a minimally invasive alternative with encouraging observational outcomes but high-quality comparative evidence versus open surgery remains limited. The MINE trial is designed to compare MRgLITT with open surgery in patients with mTLE. MINE is a prospective, randomised, open-label, blinded-endpoint trial. Eligible adults with drug-resistant TLE who complete a comprehensive presurgical evaluation and are deemed suitable for surgical treatment will be enrolled at participating centres. Participants will be randomised 1:1 (block randomisation) to receive MRgLITT or open surgery. Because of the nature of surgical interventions, participants and treating teams will not be blinded; however, outcome assessors and statistical analysts will remain blinded to treatment allocation. The primary outcome is seizure-free rate at 12 months after surgery, defined as International League Against Epilepsy (ILAE) Class 1. Secondary outcomes include: (1) broader seizure outcomes (ILAE class distribution, seizure frequency and seizure severity) assessed at 6 and 12 months; (2) cognitive and memory function assessed at 6 and 12 months; (3) postoperative safety outcomes collected from surgery through discharge and at follow-up visits at 6 and 12 months, and (4) healthcare utilisation and health-economic outcomes: length of hospital stay measured during hospitalisation; and reoperation, antiseizure medication reduction and quality of life assessed at 6 and 12 months. Safety outcomes include perioperative adverse events (AEs) and serious AEs (SAEs), collected from surgery through discharge and at follow-up visits at 6 and 12 months. This trial is powered as a non-inferiority study on the primary outcome, which based on prior data reporting 12-month seizure-free rates of 76.9% for MRgLITT and 76.0% for open surgery, the assumed absolute difference is 0.009. The non-inferiority margin was set at -0.23, informed by the minimum acceptable benefit threshold reported in patient preference research. The study has received ethics approval from the Institutional Review Board of Beijing Tiantan Hospital (KY2025-073-01). Written informed consent will be obtained from all participants prior to enrolment. Results will be disseminated through peer-reviewed publications and academic presentations. NCT06720922.

Open article ↗



2026-07-05 | Cenobamate use in super-refractory status epilepticus: A report of three cases.

Super-refractory status epilepticus (SRSE) is a neurological emergency with high morbidity and mortality. Cenobamate, a novel antiseizure medication, may be helpful in managing SRSE, but evidence is limited. This retrospective case series reports the use of cenobamate as add-on therapy in the management of three cases of SRSE. Median age at status epilepticus (SE) onset was 28 years (range 27-75). The etiologies of SRSE included one case of febrile infection-related epilepsy syndrome (FIRES), one patient with a probable genetic etiology (SCN7A variant of uncertain significance), and one case of unknown etiology. Cenobamate was introduced at a median of 27 days (range 13-103) after SE onset. Resolution of SRSE was observed after a median of 7 days (range 3-30) once cenobamate was started. Median dose of cenobamate at SRSE cessation was 25 mg/day (range 12.5-50 mg/day), and the median maintenance dose at the time of discharge was 200 mg/day (range 100-400 mg/day). Two patients died and one patient achieved functional independence. No severe medication side effects, specifically drug reaction with eosinophilia and systemic symptoms (DRESS), were observed. Cenobamate may have a role in the management of SRSE. Further studies are needed to define the optimal timing of initiation, titration strategy, and target dose of cenobamate in the context of SE.

Open article ↗



2026-06-29 | Anakinra for tocilizumab-refractory febrile infection-related epilepsy syndrome with normal IL-1β levels: a case report.

Febrile infection-related epilepsy syndrome (FIRES) is a severe epileptic encephalopathy with limited treatment options. Neuroinflammation, particularly involving IL-1β and IL-6, is a key driver. Evidence on sequential use of IL-6 receptor blockade (tocilizumab) followed by IL-1 receptor blockade (anakinra) is scarce, especially when IL-1β levels are normal. A previously healthy 6-year-old boy developed super-refractory status epilepticus six days after influenza A infection. He failed multiple antiseizure medications, anesthetics, corticosteroids, intravenous immunoglobulin, ketogenic diet, and mild therapeutic hypothermia. Cerebrospinal fluid (CSF) and serum cytokine testing showed elevated IL-6 but normal IL-1β. After initial improvement with tocilizumab, seizures relapsed. Tocilizumab retreatment and plasma exchange were ineffective. Switching to daily subcutaneous anakinra led to rapid seizure control within five days. He was weaned off anesthetics, extubated, and eventually discharged with mild cognitive dysfunction (PCPC score 2). At one-month follow-up, he had rare brief seizures and attended school. This case demonstrates that anakinra can be effective in tocilizumab-refractory FIRES even when CSF and serum IL-1β levels are normal. Anakinra should be considered as a second-line option in selected patients, regardless of IL-1β biomarker status.

Open article ↗



Access all drug discovery articles and probability of success in trials forecasts:

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Drug Discovery Landscape

7 orphan drug designations for Epilepsy syndrome, including 2 approved therapies.

7 orphan drug designations for Epilepsy syndrome, including 2 approved therapies.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

diazepam

small molecules

FDA

2016-11-10

Aquestive Therapeutics

topiramate injection

small molecules

FDA

2013-07-24

CURx Pharmaceuticals, Inc.

intravenous carbamazepine [CARNEXIV]

small molecules

FDA

2013-06-27

2016-10-07

Lundbeck LLC

diazepam auto-injector

small molecules

FDA

2013-05-30

Meridian Medical Technologies, Inc.

midazolam

small molecules

FDA

2006-05-08

UCB, Inc

Stiripentol [Diacomit]

small molecules

EMA

2001-12-05

Biocodex

Diazepam viscous solution for rectal administration [Diastat]

small molecules

FDA

1992-02-25

1997-07-29

Valeant Pharmaceuticals

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.