

Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
DAFFODIL Study Publishes Long-Term DAYBUE® Data in Rett Syndrome
Details : Daybue (trofinetide) is a inhibitor of CYP3A4, indicated for Rett syndrome, a rare neurodevelopmental disorder in 2 year and older.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
March 05, 2025
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Acadia Pharmaceuticals Submits EMA Application for Trofinetide in Rett Syndrome
Details : Daybue (trofinetide) is a inhibitor of CYP3A4, indicated for Rett syndrome, a rare neurodevelopmental disorder in 2 year and older. It is already approved by FDA for the same indication.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
January 14, 2025
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Divestment
Acadia Closes $150M Sale of Priority Review Voucher for Rare Diseases
Details : Acadia has sold its Rare Pediatric Disease Priority Review Voucher associated with Daybue (trofinetide), an approved treatment for Rett syndrome.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Undisclosed
December 11, 2024
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Divestment

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Divestment
Acadia Sells Priority Review Voucher for $150M
Details : Acadia is selling its rare pediatric disease priority review voucher for Daybue (trofinetide) is a prescription oral therapy for the treatment of Rett syndrome.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Undisclosed
November 05, 2024
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Divestment

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Acadia Pharma Announces Health Canada Approval of DAYBUE™ For Rett Syndrome
Details : Daybue (trofinetide) is a weak inhibitor of CYP3A4, which is indicated for the treatment of Rett syndrome, a rare neurodevelopmental disorder.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
October 16, 2024
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Acadia Submits Trofinetide NDA For Rett Syndrome, Accepted For Filing And Review
Details : Daybue (trofinetide) is a weak inhibitor of CYP3A4, which is indicated for the treatment of Rett syndrome, a rare neurodevelopmental disorder. It is already approved by FDA for the same indication.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
April 22, 2024
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Daybue (trofinetide) is a CYP3A4 inhibitor which helps to stimulate synaptic maturation and overcome the synaptic and neuronal immaturities that are characteristic of Rett syndrome pathophysiology.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
April 17, 2023
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Daybue (trofinetide) is a CYP3A4 inhibitor which helps to stimulate synaptic maturation and overcome the synaptic and neuronal immaturities that are characteristic of Rett syndrome pathophysiology.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
March 10, 2023
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The NDA is supported by results from the pivotal Phase 3 Lavender study evaluating the efficacy and safety of NNZ-2566 (trofinetide) versus placebo in 187 girls and young women aged 5-20 years with Rett syndrome.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
September 12, 2022
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : NNZ-2566 (trofinetide) is an investigational drug, is a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.
Product Name : Daybue
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
July 18, 2022
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
