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Therapeutic Area

Rare Disease

Therapeutic area intelligence for Rare Disease with 8 indexed intelligence signals across regulatory, clinical, competitive, and market developments.

Signals: 4
Catalysts: 4
Companies: 4

Companies

Horizon Therapeutics →

Ionis Pharmaceuticals →

Sarepta Therapeutics →

Assets

Donidalorsen →

Recent signals(4)

ClinicalJuly 5, 2026

Long-term Study on Odevixibat Effectiveness for PFIC in South Korea Initiated

A new study has been initiated in South Korea to evaluate the long-term effectiveness and safety of odevixibat in patients with PFIC.

ClinicalJuly 4, 2026

Study on Endothelial Cell Dysfunction in Pulmonary Hypertension Launched by NIH

The NIH is conducting a study to better understand pulmonary hypertension through various diagnostic tests and treatments.

ClinicalJuly 3, 2026

Ipsen initiates trial for elafibranor in Primary Sclerosing Cholangitis

Ipsen is conducting a trial to assess the efficacy and safety of elafibranor in adult patients with Primary Sclerosing Cholangitis.

ClinicalJune 28, 2026

Plerixafor vs G-CSF Trial for WHIM Syndrome: A Comparative Study

A clinical trial is underway to compare the efficacy of plerixafor against G-CSF in treating infections in patients with WHIM syndrome.

Upcoming catalysts(3)

FDA Approval

Ionis Pharmaceuticals — Donidalorsen

Approval would validate Ionis RNA platform in prophylactic rare disease setting.

August 20, 2026 · NDA/BLA

Earnings Catalyst

Horizon Therapeutics — Tepezza

Q2 earnings will reveal subcutaneous uptake and pricing dynamics post-IV cannibalization.

July 25, 2026 · Commercial

Advisory Committee

Sarepta Therapeutics — Elevidys

AdCom on broader ambulatory use carries high stakes for gene therapy revenue trajectory.

July 18, 2026 · Approved

Indications

Duchenne muscular dystrophy (ambulatory) →

Generalized myasthenia gravis (pediatric) →

Hereditary angioedema →

Rare Disease →

Thyroid eye disease (subcutaneous) →

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