Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis
(IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions.
Notarangelo, "A single-center experience in 20 patients with infantile malignant osteopetrosis
," The American Journal of Hematology, vol.
or autosomal recessive osteopetrosis (ARO) is characterized by severe osteosclerosis, pathologic fractures, hepatosplenomegaly, and pancytopenia, due to an osteoclast dysfunction that results in inadequate bone resorption.
Gamma Immunex (recombinant interferon beta 1) is an effective treatment for patients suffering from infectious diseases such as chronic granulomatous and malignant osteopetrosis
InterMune's revenues are comprised of revenues from our HCV collaboration with Roche and sales of Actimmune(R), which is approved for two indications, chronic granulomatous disease (CGD) and severe, malignant osteopetrosis
. In the United States, the company estimates that there are approximately 1,200 patients with CGD and less than 500 patients with severe, malignant osteopetrosis
Interferon beta-1a (Avonex) and interferon beta-1b (Betaseron) are approved for multiple sclerosis; interferon gamma-1b (Actimmune) reduces the frequency and severity of infections associated with chronic granulomatous disease and delays progression of malignant osteopetrosis
. With all interferons, animals given large doses have had abortions, but this does not appear to occur with doses used clinically.
Interferon beta-1a (Avonex) and interferon beta-1b (Betaseron) are approved for treating multiple sclerosis; interferon gamma-1b (Actimmune) is used to reduce the frequency and severity of serious infections associated with chronic granulomatous disease and to delay the progression of malignant osteopetrosis
Actimmune is currently marketed in the United States by InterMune for the treatment of two rare congenital diseases, chronic granulomatous disease and severe, malignant osteopetrosis
. In addition to this Phase II study in asthma, InterMune is conducting or planning to conduct multiple Phase II and Phase III clinical studies examining Actimmune's use in idiopathic pulmonary fibrosis, ovarian cancer, liver fibrosis, cryptococcal meningitis, a difficult-to-treat and life-threatening fungal infection, cystic fibrosis and atypical mycobacterial infections.
Boehringer Ingelheim and InterMune plan to immediately seek expedited EU approvals for Imukin(R) for the treatment of severe, malignant osteopetrosis
, an indication for which ACTIMMUNE(R) is already approved in the United States.
Rocket's pre-clinical pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis
Rocket's additional pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis
. Rocket is also developing an AAV-based gene therapy program for a devastating, pediatric heart failure indication, Danon disease.
ACTIMMUNE is reportedly approved by the US FDA to reduce the severity of serious infections associated with Chronic Granulomatous Disease (CGD), a genetic disorder that affects the functioning of a type of white blood cell of the immune system, neutrophils or phagocytes; as well as to slow the worsening of severe, malignant osteopetrosis
(SMO), a genetic disorder that affects normal bone formation causing the abnormal accumulation of bone material which tends to narrow the space inside bones.