Huntington’s disease (HD) is a devastating genetic disorder that robs its victims of physical and mental control. Resulting from the mutated HTT (Huntington’s) gene, it is one of the most destructive diseases known to man. There are approximately 30,000 symptomatic patients in the US and more than 200,000 at-risk of inheriting HD, usually diagnosed between the ages of 30 and 45. It impairs the patient’s ability to lead a normal life due to progressive chorea (abnormal involuntary movements) accompanied by cognitive and psychiatric problems. No treatment is available that can stop or reverse the course of HD and the average life expectancy following diagnosis is only about 10-30 years.
The partnership between Sanofi and Regeneron notched another victory with the regulatory approval of Kevzara® (sarilumab) for rheumatoid arthritis (RA). This comes after the approval of Dupixent® in March 2017 for severe atopic dermatitis. On May 23, 2017, FDA approved Kevzara®, the interleukin (IL-6) receptor blocker, for moderate to severe RA in patients with an inadequate response to disease-modifying anti-rheumatic drugs (DMARDs). This comes after the initial disappointment of receiving a Complete Response Letter from FDA in late 2016 due to deficiencies in the manufacturing process.
As per American College of Cardiology (ACC) and American Heart Association (AHA), number of patients with CHF has gone up by 800,000 over five years and median age has dropped from 63 years to 55 years in last 20 years, with male preponderance.
Cancer remains the ultimate elusive frontier in medicine. Ironically, though the war on cancer has witnessed milestones with the discovery of p53 in the 70s, RAS in the 80s, genetic revolution in the 90s and the development of personalized medicine to address molecular mutations in the last decade, cancer remains incurable.
We have had an exhilarating year indeed!! 2016 has seen the successes of Tecentriq, Cinqair,and Taltz,the failures of Revusiran, Rociletinib, and Drisapersen,and Donald Trump step into the US presidency.