Lead Product(s) : Everolimus
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Breckenridge Gains FDA Nod for Everolimus, Afinitor Disperz Generic
Details : Afinitor Disperz (everolimus) is an mTOR inhibitor small molecule drug candidate, which is currently being evaluated for the treatment of tuberous sclerosis complex.
Product Name : Afinitor Disperz-Generic
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
January 29, 2025
Lead Product(s) : Everolimus
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Amifampridine Phosphate
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lupin Gains Tentative USFDA Approval for Amifampridine Tablets
Details : Firdapse-Generic (amifampridine) is an oral, nonspecific, voltage-dependent, potassium channel blocker that is indicated to treat Lambert-Eaton myasthenic syndrome.
Product Name : Firdapse-Generic
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
March 17, 2025
Lead Product(s) : Amifampridine Phosphate
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Revakinagene Taroretcel
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Neurotech’s ENCELTO™ FDA-Approved for Macular Telangiectasia Type 2
Details : Encelto (revakinagene taroretcel) leverages is a gene therapy which delivers CNTF through an ocular implant, it is approved for the treatment of chronic retinal diseases, such as MacTel.
Product Name : Encelto
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
March 06, 2025
Lead Product(s) : Revakinagene Taroretcel
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Zinc Acetate Dihydrate
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Eton Pharmaceuticals Announces Commercial Launch of Galzin® (zinc acetate) Capsules
Details : Galzin (zinc acetate) is FDA-approved for the maintenance treatment of patients with Wilson disease who have been initially treated with a chelating agent.
Product Name : Galzin
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
March 03, 2025
Lead Product(s) : Zinc Acetate Dihydrate
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Agreement
Zevra to Sell Rare Pediatric Disease Voucher for $150M
Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C
Product Name : Miplyffa
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
February 27, 2025
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Agreement
Lead 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) : Belzutifan
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
WELIREG® (belzutifan) Receives First European Commission Approval for Two Indications
Details : Welireg (belzutifan), an oral HIF-2α inhibitor, being evaluated for the treatment of adult and pediatric patients with advanced, unresectable, or metastatic pheochromocytoma and paraganglioma.
Product Name : Welireg
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
February 18, 2025
Lead Product(s) : Belzutifan
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Eculizumab
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
CDSCO Approves AstraZeneca’s Anti-Complement Medicine Eculizumab
Details : Soliris (eculizumab) is a complement C5 inhibitor antibody which is indicated to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Product Name : Soliris
Product Type : Antibody
Upfront Cash : Inapplicable
January 17, 2025
Lead Product(s) : Eculizumab
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Eculizumab
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Teva Pharmaceutical Industries
Deal Size : Undisclosed
Deal Type : Licensing Agreement
Samsung Bioepis, Teva Partner to Commercialize EPYSQLI® in US
Details : Under the licensing agreement, Teva will be responsible for the commercialization of the product in the U.S. It is being indicated for the treatment of paroxysmal nocturnal hemoglobinuria.
Product Name : Epysqli
Product Type : Antibody
Upfront Cash : Undisclosed
January 10, 2025
Lead Product(s) : Eculizumab
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Teva Pharmaceutical Industries
Deal Size : Undisclosed
Deal Type : Licensing Agreement
Lead Product(s) : Nedosiran
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Rivfloza (nedosiran) is a once-monthly subcutaneous ribonucleic acid interference (RNAi) therapy indicated to lower urinary oxalate levels in children 9 years of age and older and adults with primary hyperoxaluria type 1 .
Product Name : Rivfloza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
October 02, 2023
Lead Product(s) : Nedosiran
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable