AI Drug Discovery for Pharma and Biotech

Drug discovery

7

drugs

With orphan designations

Overview

Sudden sensorineural hearing loss (SSNHL) is an otologic emergency characterized by rapid-onset hearing loss ≥30 dB across three contiguous frequencies within 72 hours, typically unilateral. Most cases (85–90%) are idiopathic, though viral infections, vascular compromise, and autoimmune etiologies are implicated. Diagnosis requires audiometry and exclusion of conductive causes. Immediate high-dose corticosteroids are the cornerstone of treatment, with intratympanic steroids or hyperbaric oxygen as adjuncts. Prognosis improves with early intervention, but 30–50% have permanent deficits requiring auditory rehabilitation [1][3][9][15].

Population

Affects 5–27 per 100,000 annually (~66,000 U.S. cases/year), peaking in adults aged 40–60. Incidence rises with age, with a slight male predominance [1][5][14].

Burden

Associated with tinnitus (60–70%), vertigo (30–40%), and long-term communication impairment. Unaddressed cases contribute to social isolation, cognitive decline, and economic costs (~US$1 trillion globally for all hearing loss) [4][10][14].

Therapies

  • First-line: Oral corticosteroids (e.g., prednisone 1 mg/kg) within 2 weeks of onset [3][7][11].

  • Salvage therapy: Intratympanic steroid injections for refractory cases (2–6 weeks post-onset) [7][11].

  • Adjuncts: Hyperbaric oxygen combined with steroids, particularly in early stages [11][15].

Categories: rare otorhinolaryngological diseases

Research Papers

1,646 drug discovery papers related to Sudden sensorineural hearing loss, with 4 first-in-class and 11 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

1,646 drug discovery papers related to Sudden sensorineural hearing loss, with 4 first-in-class and 11 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-01 | Intratympanic injection of human umbilical cord mesenchymal stem cell derived small extracellular vesicles for refractory sudden sensorineural hearing loss: a Phase 1 trial.

Refractory sudden sensorineural hearing loss (RSSNHL) remains a therapeutic challenge with limited effective treatment options. Human umbilical cord mesenchymal stem cell-derived small extracellular vesicles (hucMSC-sEVs) have demonstrated otoprotective and regenerative potential in preclinical studies. This Phase 1 trial evaluated the safety of intratympanic hucMSC-sEVs in patients with RSSNHL. This single-center, open-label, single-arm clinical study enrolled RSSNHL patients between June and September 2025 (ChiCTR2500103765). Participants received intratympanic hucMSC-sEVs for five consecutive days across four dose cohorts, ranging from 8 × 10⁸ to 1 × 10¹¹ particles/mL. Follow-up assessments were conducted at 1, 2, 4, and 8 weeks after treatment, during which adverse events (AEs) were recorded, and hearing outcomes were evaluated. Twelve of the thirteen enrolled patients completed the 8-week follow-up. Intratympanic administration of hucMSC-sEVs was well tolerated across all dose levels, with no dose-limited toxicity or serious AEs. The most common AEs were ear pain (5/12), ear fullness (3/12), and transient dizziness (2/12), all characteristic of intratympanic injection and resolving without intervention. In this Phase 1 study, intratympanic hucMSC-sEVs were well tolerated and showed preliminary signals of hearing improvement. Responses were observed only in patients receiving lower dose levels (8 × 10⁸ and 4 × 10⁹ particles/mL) and in those treated within a shorter disease window (≤ 22 days). These exploratory findings require confirmation in larger controlled trials. Trial Registration This study was registered on June 5,2025, at Chinese Clinical Trial Registry (ChiCTR2500103765).

Open article ↗



2026-06-30 | Via auricular ozone insufflation therapy for sudden sensorineural hearing loss: A case report

Objective: Sudden sensorineural hearing loss (SSNHL) is an otologic emergency that requires rapid diagnosis and treatment. Hyperbaric oxygen therapy is commonly recommended; however, it may be contraindicated in patients with pulmonary comorbidities. This case report presents a patient with profound unilateral SSNHL who could not receive hyperbaric oxygen therapy due to emphysema and was treated with via auricular ozone insufflation.Case Presentation: A 52-year-old male presented with same-day onset severe unilateral SSNHL. Intratympanic dexamethasone administered in five sessions provided no improvement. Hyperbaric oxygen therapy was contraindicated due to emphysema. The patient subsequently underwent ten sessions of auricular ozone insufflation at 10 gamma concentration. Post-treatment audiometry showed marked improvement, with pure-tone thresholds improving from 70–91 dB to 43–46 dB.Conclusion: Via auricular ozone insufflation may be considered as an alternative therapeutic modality in selected SSNHL patients, particularly when conventional treatments are contraindicated or ineffective. Further studies are required to standardize the use of ozone therapy in otologic practice.Keywords: sensorineural hearing loss, ozone therapy, audiogram, sudden hearing loss.

Open article ↗



2026-06-25 | Management of Sensorineural Hearing Loss in Adults: Consideration of Lifestyle Practices from a Mitochondrial Perspective.

The physiological processes of sensorineural hearing in the cochlea have high energy demands. As energy is provided mainly by mitochondria housed in cochlear cells, mitochondrial dysfunction can possibly lead to sensorineural hearing loss (SNHL). Consequently, hearing benefits can potentially be derived if measures that can potentially enhance mitochondrial health are implemented. Unhealthy lifestyle practices have led to an increased risk of developing systemic oxidative stress and chronic inflammation. These, in turn, may result in persistent mitochondrial dysfunction in the body. Together, they could possibly lead to the development of SNHL and other non-communicable chronic diseases (NCDs). Factors such as micro-nutrient deficiencies, obstructive sleep apnea, and gut dysfunction could result in mitochondrial dysfunction. On the other hand, mitochondrial function could be enhanced by interventions such as better food choices and hormetic strategies like intermittent fasting. Conventional treatments of SNHL focus on steroid therapy in acute loss and hearing rehabilitation with hearing devices in chronic loss. By integrating personalized healthy lifestyle strategies, it is postulated that the chances of hearing recovery in acute SNHL could be enhanced in some patients. In chronic SNHL, they could help to prevent/slow down its progression and possibly also the progression of concomitant NCDs. Further research is warranted to substantiate these postulations.

Open article ↗



2026-07-01 | Intratympanic injection of human umbilical cord mesenchymal stem cell derived small extracellular vesicles for refractory sudden sensorineural hearing loss: a Phase 1 trial.

Refractory sudden sensorineural hearing loss (RSSNHL) remains a therapeutic challenge with limited effective treatment options. Human umbilical cord mesenchymal stem cell-derived small extracellular vesicles (hucMSC-sEVs) have demonstrated otoprotective and regenerative potential in preclinical studies. This Phase 1 trial evaluated the safety of intratympanic hucMSC-sEVs in patients with RSSNHL. This single-center, open-label, single-arm clinical study enrolled RSSNHL patients between June and September 2025 (ChiCTR2500103765). Participants received intratympanic hucMSC-sEVs for five consecutive days across four dose cohorts, ranging from 8 × 10⁸ to 1 × 10¹¹ particles/mL. Follow-up assessments were conducted at 1, 2, 4, and 8 weeks after treatment, during which adverse events (AEs) were recorded, and hearing outcomes were evaluated. Twelve of the thirteen enrolled patients completed the 8-week follow-up. Intratympanic administration of hucMSC-sEVs was well tolerated across all dose levels, with no dose-limited toxicity or serious AEs. The most common AEs were ear pain (5/12), ear fullness (3/12), and transient dizziness (2/12), all characteristic of intratympanic injection and resolving without intervention. In this Phase 1 study, intratympanic hucMSC-sEVs were well tolerated and showed preliminary signals of hearing improvement. Responses were observed only in patients receiving lower dose levels (8 × 10⁸ and 4 × 10⁹ particles/mL) and in those treated within a shorter disease window (≤ 22 days). These exploratory findings require confirmation in larger controlled trials. Trial Registration This study was registered on June 5,2025, at Chinese Clinical Trial Registry (ChiCTR2500103765).

Open article ↗



2026-06-30 | Via auricular ozone insufflation therapy for sudden sensorineural hearing loss: A case report

Objective: Sudden sensorineural hearing loss (SSNHL) is an otologic emergency that requires rapid diagnosis and treatment. Hyperbaric oxygen therapy is commonly recommended; however, it may be contraindicated in patients with pulmonary comorbidities. This case report presents a patient with profound unilateral SSNHL who could not receive hyperbaric oxygen therapy due to emphysema and was treated with via auricular ozone insufflation.Case Presentation: A 52-year-old male presented with same-day onset severe unilateral SSNHL. Intratympanic dexamethasone administered in five sessions provided no improvement. Hyperbaric oxygen therapy was contraindicated due to emphysema. The patient subsequently underwent ten sessions of auricular ozone insufflation at 10 gamma concentration. Post-treatment audiometry showed marked improvement, with pure-tone thresholds improving from 70–91 dB to 43–46 dB.Conclusion: Via auricular ozone insufflation may be considered as an alternative therapeutic modality in selected SSNHL patients, particularly when conventional treatments are contraindicated or ineffective. Further studies are required to standardize the use of ozone therapy in otologic practice.Keywords: sensorineural hearing loss, ozone therapy, audiogram, sudden hearing loss.

Open article ↗



2026-06-25 | Management of Sensorineural Hearing Loss in Adults: Consideration of Lifestyle Practices from a Mitochondrial Perspective.

The physiological processes of sensorineural hearing in the cochlea have high energy demands. As energy is provided mainly by mitochondria housed in cochlear cells, mitochondrial dysfunction can possibly lead to sensorineural hearing loss (SNHL). Consequently, hearing benefits can potentially be derived if measures that can potentially enhance mitochondrial health are implemented. Unhealthy lifestyle practices have led to an increased risk of developing systemic oxidative stress and chronic inflammation. These, in turn, may result in persistent mitochondrial dysfunction in the body. Together, they could possibly lead to the development of SNHL and other non-communicable chronic diseases (NCDs). Factors such as micro-nutrient deficiencies, obstructive sleep apnea, and gut dysfunction could result in mitochondrial dysfunction. On the other hand, mitochondrial function could be enhanced by interventions such as better food choices and hormetic strategies like intermittent fasting. Conventional treatments of SNHL focus on steroid therapy in acute loss and hearing rehabilitation with hearing devices in chronic loss. By integrating personalized healthy lifestyle strategies, it is postulated that the chances of hearing recovery in acute SNHL could be enhanced in some patients. In chronic SNHL, they could help to prevent/slow down its progression and possibly also the progression of concomitant NCDs. Further research is warranted to substantiate these postulations.

Open article ↗



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

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

Drug Discovery Landscape

7 orphan drug designations for Sudden sensorineural hearing loss.

7 orphan drug designations for Sudden sensorineural hearing loss.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole

small molecules

FDA

2021-05-10

AudioCure Pharma GmbH

pioglitazone

small molecules

FDA

2019-12-12

Strekin AG

6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole

small molecules

EMA

2018-12-14

AudioCure Pharma GmbH

Pioglitazone hydrochloride

small molecules

EMA

2017-01-12

Regiomedica GmbH

R-azasetron besylate

small molecules

EMA

2016-11-18

Sensorion

(UDU-stereoisomer of c-UJUun UNU-terminal UkUnhibitor)

small molecules

FDA

2006-03-28

Altamira Therapeutics Inc.

H-D-Asp-D-Gln-D-Ser-D-Arg-D-Pro-D-Val-D-Gln-D-Pro-D-Phe-D-Leu-D-Asn-D-Leu-D-Thr-D-Thr-D-Pro-D-Arg-D-Lys-D-Pro-D-Arg-D-Pro-D-Pro-D-Arg-D-Arg-D-Arg-D-Gln-D-Arg-D-Arg-D-Lys-D-Lys-D-Arg-D-Gly-NH2

peptides

EMA

2005-06-16

Auris Medical Limited

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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.