HP LLC believes that an at-market commercial license for a branded, patent-protected orphan drug with seven years of statutory market exclusivity and that uniquely addresses the chronic needs of a hereditary cancer syndrome like Basal Cell Carcinoma Nevus Syndrome (BCCNS or Gorlin's syndrome
) would provide a substantial upfront payment, a portion of which might be tied to additional regulatory milestones, a significantly greater royalty, and additional significant payments tied to one-time sales milestones.
The initial link between HH signaling and human cancers was established when mutations in human PTCH1 were found to be associated with a rare hereditary disease called Gorlin's syndrome
. Patients with Gorlin's syndrome
have a high incidence of basal cell carcinoma, medulloblastoma and RMS [30-32].
HPPI will study the efficacy and safety of twice-daily dosing of SUBA-Itraconazole Oral Capsules as a potential therapy for basal cell carcinoma in patients with basal cell carcinoma nevus syndrome (BCCNS), also called Gorlin's Syndrome
The nevoid BCC syndrome or Gorlin's syndrome
is a condition with significant oral, head and neck involvement that occurs at a younger age (under 30 years) and should be recognized by dentists.
It has been explored that medullated nerve fibres are a sign of Gorlin's syndrome - a feature of which is intracranial calcification.
Since our patient's MRI also revealed the same, whether it is an early step in the pathogenesis of Gorlin's syndrome needs further evaluation.
She and her associates are conducting a phase II trial of the experimental Genentech drug GDC-0449 in patients with many BCCs from basal cell nevus syndrome (Gorlin's syndrome
), and a separate proof-of-concept study of the antifungal drug itraconazole in patients with small numbers of BCCs who do not have basal cell nevus syndrome.
I have facial disfigurement because of Gorlin's syndrome
A 10-year-old girl with Gorlin's syndrome
presented for evaluation of bilateral tonsillar lesions.
The hedgehog pathway caught the attention of cancer researchers when studies revealed that people with Gorlin's syndrome
, a hereditary condition that leaves a person highly susceptible to medulloblastoma, often lack a protein that acts as a brake on the pathway, says Berman.
NBCCS is also known as Gorlin-Goltz syndrome, Gorlin's syndrome
, and basal cell nevus syndrome.
with associated odontogenic cysts.