It’s time for my annual article regarding new medications of the year. In 2019, a variety of novel entities received FDA approval, and some drugs were approved after just a few dozen people were tested! What you are reading is syndicated worldwide, however I will only list American brand names so please ask your medication specialist to find out a medication name as it is known in your country.
Happy New Year everyone!
Vyondys 53 (Golodirsen), Rx, Injection
This medication was granted a fast track to approval for patients with Duchenne Muscular Dystrophy (DMD). Characterized by muscle deterioration and weakness, DMD occurs due to a genetic mutation and symptoms are evident in children at age 3 or 4. Jerry Lewis was a famous actor, comedian and singer who hosted telethons for Muscular Dystrophy until 2010 and would be pleased about new treatments for DMD.
Oxbryta (Voxelotor), Rx, Injection
This treats Sickle Cell Anemia which causes red blood cells to be shaped abnormally (as a crescent or “sickle” shape). This restricts blood flow and oxygen to tissues which results in pain , organ damage and inflammation. Oxbryta inhibits a pathway in the body that improves blood flow. Common side effects include headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever).
Fetroja (cefiderocol), Rx, Injection
This is a new antibiotic for Urinary Tract Infections (UTIs) that gets injected into your blood stream. It is in the category of beta-lactams so don’t take it if you’re allergic to Penicillin or Cephalosporins. Fetroja is a hospital antibiotic used to treat complicated UTIs and pyelonephritis that become life-threatening, not your regular UTI.
Reyvow (Lasmiditan), Rx, Oral
This medication is for adults with migraines (with or without aura) and should not be taken if you plan to drive. It can cause intense drowsiness. It just received FDA approval as the first and only medication in its category. It is used for acute relief and is significant because migraine pain is severe and incapacitating for many people. If you’d like more information, I’ve written a wonderful book on this called Headache Free.
Treatment may cause some adverse events and the most frequently reported ones include dizziness, fatigue, paresthesia, sedation, nausea and/or vomiting and muscle weakness.
Jeuveau (PrabotulinumtoxinA-xvfs), Rx, Injection
Think of this as the new type of Botox! It contains a botulinum derivative that temporarily erases frown lines between your eyebrows (glabellar lines). It’s pronounced Ju-Vo. Jeuveau was evaluated in 5 different clinical trials that encompassed more than 2,100 people, mainly women.
Ibsrela (Tenapanor), Rx, Oral
For Parkinson’s, this is a pill that you take twice a day, not injection. It’s used to treat Irritable Bowel Syndrome (IBS) with constipation in adults.This medication may cause serious dehydration, especially in children or elderly. The most common side effects from the initial trials are diarrhea, which sometimes may be severe, abdominal distention, excessive gas and dizziness.
Accrufer (Ferric Maltol), Rx, Oral
These are capsules of iron to help people with iron deficiency anemia. The iron drug was approved by the FDA based upon three different clinical trials. Accrufer was useful in increasing levels in the patients that took the drug twice daily. Common side effects with this medication were gas, diarrhea, constipation, stool color change, nausea, vomiting, and abdominal discomfort, bloating and pain.
Xpovio (Selinexor), Rx, Oral
This medication pronounced, X-Po-Vee-O comes as a 20mg pill and was approved in July 2019 to treat Multiple Myeloma, a blood cancer. The drug is intended to be used along with a corticosteroid (such as dexamethasone) in patients who are unresponsive to at least four prior treatments. Xpovio was fast tracked through the FDA’s approval program to allow earlier patient access and was based upon evidence from only 202 patients. It works by binding “Exportin 1” which slows cancer cell growth.
Side effects that may be experienced include reduced platelet counts (which could lead to uncontrolled bleeding), neutropenia, respiratory infections and neurological dysfunction. That last one is vague and may be better described as dizziness, fainting, confusion or other mental status changes. This drug should not be given to pregnant women.
Balversa (Erdafitinib), Rx, Oral
A medication approved to treat bladder cancer, this may be used in local, advanced or metastatic cancer whose chemotherapy did not work well enough. This med was speeded through the FDA’s approval process to make it available quickly. Thirty-two percent of 87 patients who were studied experienced either complete or partial shrinkage of their tumors. Balversa may negatively affects vision and may lead to retinal detachment. Blood phosphate levels need to be evaluated.