Measuring the Value of Therapies for Ultra-Rare Diseases

  The pricing of drugs is characterized by a tension between providing incentives to drug companies to invest in the development of innovative therapies and ensuring affordability so patients have access to needed medicines. The Institute for Clinical and Economic Review, an independent non-profit research…Continue Reading

That Other Thing in the Tax Bill Rare Disease Patients Should Worry About

The rare disease community has organized to fight a provision in the Republican tax bill that would repeal the Orphan Drug Tax Credit, an incentive that’s seen as critical to incentivizing drug developers to invest in therapies to treat small patient populations. The Orphan Drug…Continue Reading

Rare Leader: Frank Rivera, CEO of Sarcoidosis of Long Island

The Basics Name: Frank Rivera Title: President Organization: Sarcoidosis of Long Island Disease focus: Sarcoidosis, an inflammatory disease that causes inflammatory cells to form clusters known as granulomas throughout the body affecting multiple systems. Headquarters: Coram, New York How did you become involved in rare…Continue Reading

Tremeau Embraces Vioxx as Pain Treatment for Rare Diseases

Rare Daily Staff Tremeau Pharmaceuticals is seeking to bring Vioxx back to the market as a pain treatment for hemophilic arthropathy, a rare degenerative joint disease in patients with hemophilia, and has won orphan drug designation for it from the U.S. Food and Drug Administration.…Continue Reading

FDA Approves Genentech’s Hemlibra to Treat Hemophilia A

Rare Daily Staff The U.S. Food and Drug Administration Genentech approved Hemlibra to prevent or reduce the frequency of bleeding episodes in children and adults with hemophilia A who have developed inhibitors for factor VIII replacement therapies.  Hemophilia A is an inherited disorder in which…Continue Reading

Khondrion Reports Positive Preliminary Data on Mid-Stage Trial in MELAS and MIDD Syndromes

Rare Daily Staff Khondrion presented promising results from a mid-stage exploratory study of KH176, its experimental therapy to treat MELAS and MIDD syndromes, two rare genetic mitochondrial disorders. MELAS is an acronym for mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes, a condition that is multiple…Continue Reading

X4 Pharmaceuticals Raises $27 Million in Series B Financing

Rare Daily Staff X4 Pharmaceuticals said it completed a $27 million series B financing that will be used to fund a pivotal study of its lead clinical candidate X4P-001, a treatment for the rare primary immunodeficiency disease WHIM syndrome. WHIM is an acronym for warts,…Continue Reading

Failed COPD Drug Shows Promise as Treatment for Rare Bone Disease

Rare Daily Staff A drug Roche had unsuccessfully sought to develop as a treatment for chronic obstructive pulmonary disease has shown promise in preclinical testing as a potential treatment for a rare genetic bone disease. Researchers at Sanford Burnham Prebys Medical Discovery Institute led a…Continue Reading

Shire Names Thomas Dittrich as CFO and Director

Rare Daily Staff Rare disease drug company Shire said it named Thomas Dittrich as its CFO and as a member of its board of directors. He replaces Jeff Poulton, who in August announced he would step down at the end of the year to join…Continue Reading

Lessons from Pitching Venture Capitalists

Stories abound about plucky entrepreneurs financing their startups, but it’s unusual to get trench-view insights from founders who have slogged their way through endless pitches along Sand Hill Road. Ethan Perlstein, founder and CEO of the rare disease drug discovery company Perlara, shared what he…Continue Reading