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Indication

Rare Disease

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

Signals
8
Catalysts
0
Assets
6

Relevant assets

Ipsen →
Launch →
NDA217379 →
NULIBRY →
Plerixafor →
SKYCLARYS →

Relevant companies

Biogen →
FLEX PHARMA, LLC →
G-CSF →

Recent signals(8)

RegulatoryAugust 17, 2026

FDA Accepts Supplemental Application for Biogen's SKYCLARYS

The FDA has accepted a supplemental application (NDA216718) for Biogen's SKYCLARYS, containing omaveloxolone.

RegulatoryAugust 12, 2026

FLEX PHARMA receives FDA application approval for NDA217379

FLEX PHARMA, LLC has received application approval (AP) for NDA217379 from the FDA, with a standard review priority. This approval pertains to a product targeting a rare neurological condition, an area characterized by limited competition and significant unmet medical needs.

RegulatoryAugust 10, 2026

FDA Accepts Supplement Application for NULIBRY by Sentyln Therapies

The FDA has accepted the supplemental application NDA214018 for NULIBRY, an enzyme replacement therapy containing fosdenopterin hydrobromide, submitted by Sentyln Therapies.

ClinicalAugust 9, 2026

Launch of Natural History Study for Niemann-Pick Disease Type C to Build Comprehensive Database

A new natural history study aims to establish the first and largest genomic and phenotypic database for Niemann-Pick Disease Type C.

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

No catalysts indexed for this entity yet.

Therapeutic areas

Enzyme Replacement Therapy →
Hepatology →
Immunology →
Neurology →
Pulmonary Hypertension →
Rare Disease →

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