The trees have shrugged off the last of their leaves, allowing twinkle lights to be suspended from their branches. Stockings are hung. Trees are up. And while we may be lulled into thinking that it is going to be a quiet time of the year, in fact, everyone is gearing up to get everything done before the year end and the pace is a little frenetic and we might be busier than we want to be which means that last week, some of us didn’t even get the Weekly Roundup out and can barely get it done today!
That said, here it is and here is some of what happened this week and a little bit of last:
- Immune Stimulating Therapy Approved – FDA announced this week that it was approving Empliciti in combination with two other treatments for the treatment of multiple myeloma. FDA stated that Empliciti works to activate the body’s own immune system to attack and kill multiple myeloma cells. The two other treatments with which Empliciti are to be administered are Revlimid and dexamethasone and is the second approval this month for multiple myeloma which the National Cancer Institute estimates will have over 26,000 cases diagnosed this year. Empliciti received breakthrough therapy status from FDA along with orphan drug designation and priority review. A company press release can be found here.
- FDA OKs Marketing of Cooling Cap for Those in Chemotherapy – Ok, when I first saw this I misread the FDA headline on the press release to say that “FDA Allows Marketing of Cool Cap…” But perhaps that should be the headline. For many hair loss can be an unpleasant side effect while undergoing chemotherapy. This week FDA ok’d a product that is meant to minimize that side effect. The way the cap is meant to work is that it circulates a cooled liquid through the cap that reduces blood flow that in turn causes the chemotherapy to have less of an impact on the hair follicles. The FDA press release states that two-thirds of women in studies using the cap reported less hair loss when compared to women who did not use the cap. The release does not state how long one must wear the cap, but did say that common side effects included discomfort to the head, neck and shoulders and chills. The product was approved through FDA’s de novo classification process which is a pathway for low to moderate risk devices that are novel and not substantially equivalent to legally marketed devices.
- Two Orphan Drug Approvals – In a year that has to be a banner year for orphan drug approvals, two new ones were added this week. First was the approval of Kanuma (sebelipase alpha) for the treatment of lysosomal acid lipase (LAL) deficiency. This condition can cause a harmful build up of fats within cells that can lead to liver and cardiovascular complications and in its more severe form, patients do not survive the first year of life. The therapy provides a protein that replaces the function disrupted by the genetic disorder that causes the deficiency and is manufactured using genetically engineered chickens. In another approval this week, FDA announced action this week to ok the first recombinant von Willebrand factor for the treatment of bleeding episodes caused by protein deficiency that causes von Willebrand disease and affecting about 1 percent of the population.
That’s it for me this week. Have to get back to the rush of the season. In the meantime, have a great weekend.