January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
Market Exclusivity Bears Little Relationship to Increase in Rare Disease Drugs, Finds Study
May 13th 2018While the number of drugs approved for rare diseases has increased significantly since the passage of the Orphan Drug Act of 1983, questions linger about whether is connected with innovation in treatments for rare diseases, especially given the fact that patent protection for new products often extends well beyond the 7 years granted to orphan drugs.
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The Rise of Orphan Drugs for Rare Diseases
May 7th 2018Since the Orphan Drug Designation program was passed in 1983, there have been over 600 orphan drug approvals, compared to less than 10 the decade before, said Stephen Jung, PharmD, BCPS, manager, drug information, MedImpact Healthcare Systems, during a session at the Academy of Managed Care Pharmacy’s Managed Care & Specialty Pharmacy Annual Meeting.
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Dr Aimee Tharaldson on Recently Approved Specialty Drugs, Upcoming Approvals to Watch
May 5th 2018Aimee Tharaldson, PharmD, a senior clinical consultant in Emerging Therapeutics for Express Scripts, discusses specialty drugs that have recently been approved, and which we will likely see approved in the remainder of the year.
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Research Provides Insight Into Cardiac-Related Mortality in Huntington Disease
May 1st 2018Huntington disease (HD) is an incurable, inherited neurological disorder caused by the mutant Huntingtin gene, which produces a mutant form of Huntingtin protein (mHTT). In addition to creating the profound neurological impacts of HD, the mHTT protein also impairs other organ systems, and new research, published in Cell Reports, suggests that the protein may play a role in cardiac-related mortality in patients with HD.
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Patient-Reported Outcome Measures Are Key to Understanding Rare Diseases
April 29th 2018Rare disease can significantly reduce patients’ quality of life, and the use of patient-reported outcome measures (PROMs) can be key to helping researchers understand how diseases and treatments impact quality of life and symptoms, according to a review newly published in the Orphanet Journal of Rare Diseases.
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Positive Interim Data Published for Transfusion-Dependent Β-Thalassemia Gene Therapy
April 24th 2018Drug company bluebird bio, Inc, has announced the publication of positive interim data for its gene therapy to eliminate or reduce chronic blood transfusions in patients with transfusion-dependent β-thalassemia.
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FDA Committee in Favor of Cannabidiol to Treat Two Rare Seizure Disorders
April 22nd 2018The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has unanimously recommended approval of the New Drug Application for GW Pharmaceuticals’ proposed cannabidiol oral solution for the adjunctive treatment of seizures associated with 2 rare disorders, Lennox-Gastaut syndrome and Dravet syndrome, in patients 2 years and older.
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Price Tag for Newly Approved Crysvita Is "Responsible," Says PBM Express Scripts
April 18th 2018Taking the rebates that it expects to negotiate into consideration, Ultragenyx said that burosumab will cost approximately $160,000 per patient per year for children and $200,000 per patient per year for adults, depending on body weight. The company said that it does not plan to increase costs in the early years of the drug’s availability.
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As New Duchenne Therapy Enters Phase 1 Trial, Advocacy Groups Seek Better Access to Existing Drug
April 15th 2018Pfizer has dosed its first patient in a phase 1 clinical trial of an investigational gene therapy for the treatment of patients with Duchenne muscular dystrophy, a genetic disease that results in the absence of dystrophin, a protein that helps to keep muscle cells intact.
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Discovery About Rare Bone Disease May Also Hold Clues About Bone Health
April 12th 2018National Institutes of Health researchers have uncovered a genetic basis of a rare bone disease known as melorheostosis and said the discovery could offer potential treatment targets, provide clues about bone development, and lead to insights about fracture healing and osteoporosis.
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Pediatric Sickle Cell Disease Drug Gains European Orphan Designation
April 8th 2018Sancilio Pharmaceuticals announced that it has received the European Medicines Agency’s Orphan Designation for its SC411 (which it plans to market as Altemia), a proposed treatment for sickle cell disease in pediatric patients, in the European Union. The FDA granted the proposed drug a similar designation—the Rare Pediatric Disease designation—in 2017.
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Siponimod Reduced Progression in Secondary Progressive Multiple Sclerosis in a Phase 3 Trial
April 1st 2018Results of a double-blind, randomized, phase 3 trial, published last week in The Lancet, show that siponimod—a selective sphingosine 1-phosphate receptor modulator—slowed the progression of disability in secondary progressive multiple sclerosis.
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Boys Don't Get HPV Vaccination Because Doctors Don't Recommend It, Study Finds
March 26th 2018Uptake for the human papillomavirus (HPV) vaccination has never reached CDC targets. Minority children from lower-income households are more likely to get the vaccination than white children from higher-income households, according to the study author.
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ICER Review Finds Hemlibra Lowers Costs and Improves Outcomes in Hemophilia A
March 25th 2018Recently, the Institute for Clinical and Economic Review (ICER) released an evidence report that investigated the comparative clinical effectiveness and value of emicizumab (marketed as Hemlibra, developed by Genentech) in the treatment of hemophilia A.
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Price and Clinical Factors Impact Canadian Orphan Drug Recommendation Rates
March 11th 2018In Canada, there exists no distinct regulatory or reimbursement pathway for orphan drugs. Instead, these products undergo the same review processes as any other drug. When a medicine is submitted for review, the Common Drug Review examines the clinical and economic evidence, and its expert committee issues a nonbinding positive or negative recommendation for listing on publicly funded drug plans.
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CSPG4 Could Make CAR T-Cell Therapy a Reality in Treating Glioblastoma
March 4th 2018Researchers have identified a molecular target that could allow chimeric antigen receptor (CAR) T-cell therapy to be used in treating patients with glioblastoma. Although the heterogeneous expression of tumor-associated antigens limits the efficacy for CAR-redirected T cells for the treatment of glioblastoma, chondroitin sulfate proteoglycan 4 (CSPG4), a cell surface type 1 transmembrane protein, is highly expressed in a majority of glioblastoma specimens with limited heterogeneity.
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Kazia Gains Orphan Designation for Investigational Glioblastoma Treatment
February 25th 2018Kazia Therapeutics Limited, an oncology-focused biotechnology company, announced on Friday that it has received the FDA’s Orphan Drug Designation for its investigational drug, GDC-0084, for the treatment of glioblastoma multiforme, the most aggressive form of primary brain cancer.
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FDA Approves Symdeko to Treat the Underlying Cause of Cystic Fibrosis
February 18th 2018Drug maker Vertex has announced that the FDA has approved tezacaftor/ivacaftor and ivacaftor (Symdeko) for the treatment of the underlying cause of cystic fibrosis (CF) in patients aged 12 or older who have 2 copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, or who have at least 1 genetic mutation that is responsive to treatment with tezacaftor/ivacaftor.
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Study Finds Merkel Cell Carcinoma Incidence Has Increased Since 2000
February 17th 2018Merkel cell carcinoma (MCC) is a rare disease, but it's getting less rare. Study results presented at the American Academy of Dermatology 2018 Annual Meeting found that cases of MCC increased 95% between 2000 and 2013
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New Guidelines for Rare Disease Advocacy Groups to Navigate Interactions With Pharma
January 28th 2018Only 5% of rare diseases have treatments, but approximately half have patient advocacy organizations; these groups have the potential to play major roles in positively influencing research and development of drugs, clinical trials, and regulations by championing funding and awareness efforts, forming connections between experts and drug developers, advocating for changes to regulation to expedite research, facilitating patient registries, or other initiatives.
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FDA Approves First Radioactive Drug for Rare Type of GI Cancer
January 27th 2018The FDA approved the first radioactive drug, or radiopharmaceutical, to treat a rare type of cancer that affects the pancreas or gastrointestinal tract called gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Lutathera (lutetium Lu 177 dotatate) is cleared for adult patients with somatostatin receptor-positive GEP-NETs.
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Study Finds Prior Authorization Mandates for PCSK9 Drugs Raise Questions of Access
January 20th 2018Researchers were especially concerned about barriers for patients with familial hypercholesterolemia, whose needs would seem clear cut but who nonetheless faced costly hurdles, such as genetic testing.
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Proposed Drug to Treat Oculopharyngeal Muscular Dystrophy Gains Orphan Drug Designation
January 19th 2018Benitec Biopharma, an Australia-based drug developer, has received the FDA’s Orphan Drug designation for BB-301, an investigational drug for the treatment of oculopharyngeal muscular dystrophy.
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