Rare Disease News and Research

RSS

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Repligen Corporation announced today that the Office of Orphan Products Development of the Food and Drug Administration has granted orphan drug designation to RG2833, a selective histone deacetylase 3 inhibitor for the treatment of Friedreich's ataxia. Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.

24 May 2010

OnePath Gaucher Application available for patients with Type 1 Gaucher disease

Shire plc, the global specialty biopharmaceutical company, today announced the availability of the OnePath Gaucher Application. The first and only application developed specifically for patients in the U.S. with Type 1 Gaucher disease, it is free and ready for download at the iTunes Application store.

24 May 2010

Grifols reaches agreement with Pharmalink to acquire PPS drug development project

Today, Grifols SA announced that it has reached an agreement with the Swedish company Pharmalink AB to acquire various forms of intellectual property associated with the treatment of post-polio syndrome. The acquisition is expected to be finalized in the next few weeks and will include documentation, know-how, and Swedish regulatory approvals under the trade name Xepol.

20 May 2010

FDA grants orphan drug designation to pralatrexate for treatment of advanced TCC of urinary bladder

Allos Therapeutics, Inc. announced today that the U.S. Food and Drug Administration has granted orphan drug designation to pralatrexate for the treatment of advanced or metastatic transitional cell carcinoma of the urinary bladder, a form of bladder cancer. The Company is currently investigating pralatrexate in a Phase 2 clinical study in patients with advanced or metastatic relapsed TCC of the urinary bladder.

18 May 2010

FDA accepts for filing Sigma-Tau Pharmaceuticals' cysteamine hydrochloride ophthalmic solution NDA

Sigma-Tau Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has accepted the filing of the company's new drug application (NDA) for its cysteamine hydrochloride ophthalmic solution, an investigational therapy for the treatment of corneal cystine crystals in patients with cystinosis. The FDA also granted the NDA Priority Review status, a designation given to drugs that may provide major advances or a treatment where no adequate therapy exists.

18 May 2010

Duchenne Muscular Dystrophy Awareness Week declared to honor work of PPMD

The Pennsylvania House of Representatives has passed a resolution declaring the week of May 24, 2010 as "Duchenne Muscular Dystrophy Awareness Week" to help honor the work of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy.

15 May 2010

NIAMS, NINDS announce 5-year, $7.5M natural history study of Duchenne muscular dystrophy

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Institute of Neurological Disorders and Stroke (NINDS) are launching a five-year, $7.5 million natural history study of Duchenne muscular dystrophy (Duchenne), a degenerative genetically-linked neuromuscular disease. The study aims to validate non-invasive approaches to monitor the progression and treatment of Duchenne, and holds potential to facilitate the development of promising new therapies for people with the disease.

10 May 2010

Aeterna Zentaris granted FDA orphan-drug designation for AEZS-108 in ovarian cancer

Aeterna Zentaris Inc. a late-stage drug development company specialized in oncology and endocrinology, today announced that it has received from the U.S. Food and Drug Administration (FDA), orphan-drug designation for AEZS-108, its doxorubicin targeted conjugate compound, for the treatment of ovarian cancer. AEZS-108 is currently in a Phase 2 trial in advanced ovarian and advanced endometrial cancer in Europe.

6 May 2010

NxStage Medical first-quarter net revenue increases 20% to $40.4 million

NxStage Medical, Inc., a leading manufacturer of innovative dialysis products, today reported financial results for the first quarter of 2010 ended March 31, 2010, with total revenue above the top end of its guidance range.

5 May 2010

Triple-S Management first-quarter total consolidated operating revenues increase 9.8% to $519.1 million

Triple-S Management Corporation, the largest managed care company in Puerto Rico, today announced consolidated operating revenues of $519.1 million and operating income of $16.4 million for the three months ended March 31, 2010. Net income of $11.2 million, or $0.38 per diluted share, includes an after tax net gain of $0.4 million, or $0.01 per share, in net realized and unrealized gains on investments and derivatives.

5 May 2010

Cadence Pharmaceuticals resubmits OFIRMEV NDA

Cadence Pharmaceuticals, Inc. announced today that it has resubmitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational product candidate, OFIRMEV™ (acetaminophen) injection, for the treatment of pain and fever in adults and children.

5 May 2010

LightLab Imaging granted FDA clearance for C7-XR Imaging System and C7 Dragonfly Imaging Catheter

LightLab Imaging, Inc., the pioneer and leader in the development of Optical Coherence Tomography (OCT) technology for vascular and other imaging applications, has received clearance from the U.S. Food and Drug Administration (FDA) for the C7-XR Imaging System and companion C7 Dragonfly Imaging Catheter.

5 May 2010

Micromet reports total revenues of $6.3 million for first-quarter 2010

Micromet, Inc., a biopharmaceutical company focused on the development and commercialization of next-generation antibodies for the treatment of cancer, today announced its financial results for the first quarter ended March 31, 2010.

5 May 2010

BELLUS Health to discontinue NC-503 diabetes development program

BELLUS Health announced today that it will discontinue its NC-503 diabetes development program following the results of the recently completed Phase IIa clinical proof-of-concept study evaluating NC-503 (eprodisate disodium) as a treatment for Type 2 diabetes. The study did not meet its primary efficacy endpoint.

5 May 2010

Synageva BioPharma seeks FDA orphan drug designation for SBC-102 enzyme replacement therapy

Synageva BioPharma Corp., a privately held biopharmaceutical company, today announced its filing with the U.S. Food and Drug Administration (FDA) requesting orphan drug designation for its program, SBC-102. This program is an enzyme replacement therapy in preclinical development to treat lysosomal acid lipase (LAL) deficiency, also known as Wolman disease or cholesteryl ester storage disease (CESD), a condition for which there is currently no effective treatment.

4 May 2010

Neural stem cell transplantation promising for Batten disease

Neuronal Ceroid Liposfuscinosis (NCL), often referred to as Batten disease, is a rare and fatal neurodegenerative disorder that afflicts infants and young children. NCL is caused by a deficiency of a lysosomal enzyme, and is a relatively rare disease, affecting an estimated 2 to 4 of every 100,000 babies born in the U.S. Although NCLs are classified as rare diseases, they often affect more than one child in families that carry the defective gene, and tragically, are always fatal.

4 May 2010

ImmunoCellular Therapeutics seeks FDA orphan drug status for ICT-107 dendritic-cell based vaccine for GBM

ImmunoCellular Therapeutics announced today it has filed an orphan drug application with the U.S. Food and Drug Administration (FDA) for its cancer treatment candidate ICT-107. ICT-107 is a dendritic-cell based cancer vaccine product which targets glioblastoma multiforme (GBM), the most prevalent, aggressive and malignant form of primary brain tumors.

3 May 2010

DARA Therapeutics collaborates to advance clinical study of KRN5500 for CIPN in cancer patients

DARA BioSciences, Inc. announced today that on April 26, 2010, DARA Therapeutics, a subsidiary of DARA BioSciences, and the Division of Cancer Prevention, National Cancer Institute (NCI), National Institutes of Health (NIH), entered into a Clinical Trials Agreement to advance the clinical study of KRN5500 for the treatment of Chemotherapy Induced Peripheral Neuropathy (CIPN) in patients with cancer. Under the terms of the collaboration, NCI will fund the studies and DARA will supply KRN5500 and placebo.

28 Apr 2010

Alexion Pharmaceuticals reports total revenues of $117.6M from net product sales of Soliris

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter ended March 31, 2010.

22 Apr 2010

Lotus Pharmaceuticals' asthma drug Laevo-Bambutero to receive Chinese SFDA Fast Track Approval

Lotus Pharmaceuticals, Inc., a growing developer, manufacturer and seller of medicine and drugs in the People's Republic of China reported that its asthma drug Laevo-Bambutero will receive the China's State Food & Drug Administration's (SFDA) Fast Track Approval to enter clinical trials. The SFDA's Drug Review and Evaluation Center told the Company during recent consultations at the end of the Company's IND that formal approval would be granted by the Beijing office of the SFDA.

21 Apr 2010

Rare Disease News and Research

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

OnePath Gaucher Application available for patients with Type 1 Gaucher disease

Grifols reaches agreement with Pharmalink to acquire PPS drug development project

FDA grants orphan drug designation to pralatrexate for treatment of advanced TCC of urinary bladder

FDA accepts for filing Sigma-Tau Pharmaceuticals' cysteamine hydrochloride ophthalmic solution NDA

Duchenne Muscular Dystrophy Awareness Week declared to honor work of PPMD

NIAMS, NINDS announce 5-year, $7.5M natural history study of Duchenne muscular dystrophy

Aeterna Zentaris granted FDA orphan-drug designation for AEZS-108 in ovarian cancer

NxStage Medical first-quarter net revenue increases 20% to $40.4 million

Triple-S Management first-quarter total consolidated operating revenues increase 9.8% to $519.1 million

Cadence Pharmaceuticals resubmits OFIRMEV NDA

LightLab Imaging granted FDA clearance for C7-XR Imaging System and C7 Dragonfly Imaging Catheter

Micromet reports total revenues of $6.3 million for first-quarter 2010

BELLUS Health to discontinue NC-503 diabetes development program

Synageva BioPharma seeks FDA orphan drug designation for SBC-102 enzyme replacement therapy

Neural stem cell transplantation promising for Batten disease

ImmunoCellular Therapeutics seeks FDA orphan drug status for ICT-107 dendritic-cell based vaccine for GBM

DARA Therapeutics collaborates to advance clinical study of KRN5500 for CIPN in cancer patients

Alexion Pharmaceuticals reports total revenues of $117.6M from net product sales of Soliris

Lotus Pharmaceuticals' asthma drug Laevo-Bambutero to receive Chinese SFDA Fast Track Approval

Achieving and maintaining ISO 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

Latest News