There’s A Gap In Treatment For Sickle Cell Disease. This Small Biotech Company Might Fill It.
There are very few treatments for the disease and a cure through gene-editing is years off. But Global Blood Therapeutics has created a drug that could offer hope to those with sickle cell.
Stat:
A Sickle Cell Drug Reaches A Critical Readout. Here’s What You Need To Know
People with sickle cell disease have few effective treatment options. The FDA has only approved two drugs — 20 years apart — to treat the inherited blood disorder. Efforts to develop a cure for sickle cell disease using gene therapy or genome-editing have shown early promise but are still years away from being proven. A new and novel medicine from Global Blood Therapeutics (GBT), a small biotech based south of San Francisco, could soon fill that sickle cell treatment gap. A late-stage clinical trial is nearing an interim but important readout before the end of the second quarter. (Feuerstein, 5/4)
In other pharmaceutical news —
The Wall Street Journal:
Cardinal Health Shares Plunge Amid Inventory, Generic-Drug Price Problems
Cardinal Health Inc. reported a sharp decline in profit in its last quarter and lowered its full-year outlook following inventory write-downs in its overseas businesses and a sharper-than-anticipated decline in generic drug prices. Shares of Cardinal Health, a drug and medical supplies wholesaler, plunged 18% to $53 on Thursday, while the S&P 500 fell 1.3%. Cardinal’s comments on drug-price deflation appeared to raise concerns about competing drug wholesalers, with shares of AmerisourceBergen Corp. falling 5% to $87.61 and shares of McKesson Corp. falling 5.4% to $146.35. (Walker, 5/3)
Kaiser Health News:
For Shame: ‘Pharma Bro’ Shkreli Is In Prison, But Daraprim’s Price Is Still High
It was 2015 when Martin Shkreli, then CEO of Turing Pharmaceuticals and the notorious “pharma bro,” jacked up the cost of the lifesaving drug Daraprim by 5,000 percent. Overnight, its price tag skyrocketed from $13.50 a pill to $750. The move drew criticism from all corners. Congress hauled Shkreli in for questioning on television. Media outlets shamed the practice. The Pharmaceutical Research and Manufacturers of America (PhRMA), the powerful trade group for branded drugs, distanced itself, saying Turing “does not represent the values of @PhRMA” and kicked off a campaign it described as “more lab coat, less hoodie.” (Luthra, 5/4)
Bloomberg:
Glaxo's Advair Is The $100 Billion Asthma Drug That Won't Die
Since 2001, Advair has helped children and adults with chronic lung diseases while raking in about $100 billion in sales for Glaxo along the way. Yet eight years after the patent on the drug itself expired and two years since its companion Diskus inhaler lost exclusivity, it remains untouched by competition from generics in the U.S. -- and patients like Bassett pay the price. ...After a series of setbacks for manufacturers of copycat drugs, the nation’s health-care system and many of the 25 million Americans with asthma remain burdened with the costs of Advair. (Paton, 5/4)
