It was hard to miss the coverage this week of new guidelines from the National Institute of Allergies and Disease (NIAID) – published January 5 in J Allergy Clin Immunol – suggesting that early introduction of peanuts into infants’ diets may prevent a potentially life-threatening allergy from developing. Peanut allergies affect about 3 million Americans. The guidelines suggest introducing age-appropriate, peanut-containing foods 1) to infants with severe eczema, egg allergies or both at 4-6 months of age; 2) to infants with mild to moderate eczema around 6 months of age; and 3) freely to infants with no eczema or food allergy.
PCSK9 battle line
On January 3, a federal judge upheld an earlier verdict against Sanofi and Regeneron for infringing on Amgen’s PCSK9 patents and banning the sale of the two companies’ PCSK9 inhibitor Praluent© (alirocumab). The ban – a major victory for Amgen and its PCSK9 inhibitor, Repatha© (evolocumab) – takes effect 30 days after the recent ruling, to allow Sanofi and Regeneron time to ask an appeals court to lift it, which the companies say they’ll do. Currently indicated for people with genetically-caused severely high blood cholesterol, PCSK9 inhibitors are a relatively new class of drugs that markedly reduce levels of low-density lipoproteins (LDLs, the “bad” cholesterol) by 50-70% even in patients already taking a statin drug.
Antisense finally making cents?
A recent article in The Scientist describes how, after decades of research fits and starts, a number of promising oligonucleotide antisense therapies appear to be on the horizon. The basic idea of antisense therapy is to synthesize a complementary strand of nucleic acid (DNA, RNA or a chemical analogue) that binds to messenger RNA (mRNA) produced by a defective gene sequence that causes a disease, and effectively turn the gene off. Or, the complementary strand might be designed to “correct” the content of defective mRNA. Synthetic oligonucleotides are so named because they are short chains of just 10-30 nucleotides (based on the Greek word “oligos” for few or scanty).
Currently, four anitsense oligonucleotides are approved, the most recent – lauded by Forbes and others as a “Christmas surprise” – on December 23 for Biogen/Ionis’ Spinraza™ (nusinersen), making it the first approved treatment for spinal muscular atrophy, the leading genetic cause of infant death. The Scientist article profiles other nucleotide-based therapies in Phase 3 trials, e.g., alicaforsen (Ionis/Atlantic Healthcare) for a specific inflammatory bowel condition, aganirsen (Gene Signal) for eye neovascularization, and others. NEJM (January 5) has a report of a Phase 1 study and a Perspective piece about PCSK9-targetd oligonucleotide therapy as a new class of drugs that are highly potent in reducing cholesterol with just one injection every 6 months vs. every 2 to 4 weeks for current PCSK9 inhibitors.
Read More about oligonucleotide therapies: NEJM
Ebola vaccine successful
On December 23, the World Health Organization confirmed in a press release that the newly developed rVSV-ZEBOV (Merck, Sharpe & Dohme) is the first highly protective vaccine against the deadly virus. Of the nearly 6,000 individuals in Guinea who received the vaccine in the trial, none suffered an Ebola infection. The trial was reported in The Lancet.
FDA new drug approvals down in 2016
As reported in Fortune and Fierce Biotech, FDA approved only 19 new drugs in 2016, compared to 45 in 2015, one of the highest since 1996. The agency’s John Jenkins said reasons include early approvals in 2015 that weren’t supposed to be processed until 2016; fewer applications in 2016; more rejections than usual by the agency, due to manufacturing concerns; and an increase in applications that resulted in a complete response letter.
Medical and health policy perspectives
Not for geeks only: Top technical advances of 2016
The Scientist profiled these advances, which include: