3
ALL3
Mirum PharmaceuticalsYear
3
ALL1
20251
20241
2023DEALS // DEV.
3
ALL3
DevelopmentsCountry
3
ALL3
U.S.A3
ALL3
InapplicableTherapeutic Area
3
ALL2
Genetic Disease1
Rare Diseases and DisordersStudy Phase
3
ALL1
Approved FDF2
Phase IIIDeal Type
3
ALL3
InapplicableProduct Type
3
ALL3
Other Small MoleculeDosage Form
3
ALL3
Oral Tablet, Film CoatedLead Product
3
ALL3
Chenodeoxycholic AcidTarget
3
ALL3
CholesterolLead Product(s) : Chenodeoxycholic Acid
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Disease
Details : Ctexli (chenodiol) is an oral cholesterol synthesis inhibitor which is being evaluated for the treatment of cerebrotendinous xanthomatosis.
Product Name : Ctexli
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
February 21, 2025
Lead Product(s) : Chenodeoxycholic Acid
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Chenodeoxycholic Acid
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Mirum Submits New Drug Application to FDA for Chenodiol for the Treatment of CTX
Details : Chenodal (chenodeoxycholic acid) is an oral cholesterol synthesis inhibitor which is being evaluated for the treatment of cerebrotendinous xanthomatosis.
Product Name : Chenodal
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
June 28, 2024
Lead Product(s) : Chenodeoxycholic Acid
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Chenodeoxycholic Acid
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Chenodiol (chenodiol) tablets is a naturally occurring bile acid, which is investigated for the treatment of adult patients with cerebrotendinous xanthomatosis.
Product Name : Chenodal
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
February 10, 2023
Lead Product(s) : Chenodeoxycholic Acid
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable