Tag Archives: FDA

Remember to Take Kuvan, PKU Formula with RxmindMe

RxmindMe IconIt is probably safe to assume that most, if not all, PKU patients do not want their genetic disorder to define who they are as a person. Instead, we attend school, hold down jobs, and engage in hobbies just like people without PKU. There is no denying, however, that PKU patients must juggle these activities while also managing their low-protein diets. Well, as the popular saying goes, “there’s an app for that!”

The app, which is called RxmindMe, is available for free on iTunes and includes several different types of reminders, including:

  • Every “x” number of days
  • Hourly
  • Daily
  • Weekly
  • On specific days (like say the 15th of every month)
  • Spontaneous (recording medication that was taken as needed as opposed to scheduled)
  • Prescription quantity (to remind you when it is time to order a refill)

Users can also export their prescription data into Excel or email their prescription history directly from their phone. This seems like an awesome feature for when you find yourself in a doctor’s office and have to list all the current medications you are taking. It is also nice to know that all data within RxmindMe is connected to the U.S. Food and Drug Administration’s database so that you can search for your drug(s) and perhaps investigate possible side effects or hazardous drug interactions.

RxmindMe, Kuvan Reminder, PKU ReminderPKU patients can use RxmindMe to enter reminders for taking Kuvan, formula or even non-PKU meds like allergy pills, antacids and contraception. There’s no limit to how many reminders you can enter. For those parents with PKU kids, getting them involved with creating the reminders on the iPhone or iPad might even encourage them to be more independent in their treatment.

Finally, for those of you into social media, follow RXmindMe on Twitter—their Twitter handle, or name, is @RxmindMe—so you can get updates and tips for using the application.




Filed under Formula, Kuvan, Lo-pro Diet Management, Technology

Maternal PKU and Sapropterin

This past February, the National Institutes of Health (NIH) hosted a scientific review conference on Phenylketonuria to discuss the “state of the science and future research needs.” The event was free and open to the public, but even if you could not cover the cost of travel and accommodations, the two-day event was also broadcasted live via webcast. I managed to log-on during my lunch break and listened intently to a session on PKU and Pregnancy. To my surprise, much of the PKU and Pregnancy presentation focused on Sapropterin and its use during maternal PKU (Sapropterin is the generic name for Kuvan, the first prescription drug approved by the U.S. Food and Drug Administration to lower blood phe levels in PKU patients).

To be clear, Kuvan is not recommended for maternal PKU unless the benefits outweigh the risks. That is because when ranked on the FDA’s pregnancy classification of drugs, Sapropterin is considered a category C drug since it is still unclear whether Sapropterin crosses the human placenta.

Research presented at the NIH conference confirmed that in some rare instances, Sapropterin had been prescribed to pregnant PKU patients, but only when the woman could not achieve dietary control at the start of her pregnancy. As early as 2005, Sapropterin was the focus of a study led by medical pioneer and PKU advocate Dr. Richard Koch. The patient was responsive to Sapropterin and during her pregnancy, she continued the medication at 40, 60, and 100 mg per day in the first, second, and third trimesters. Used in combination with low-protein food, optimal phe levels were achieved without any nausea and vomiting. More importantly, the woman’s child was born normal. Four years later, another study conducted by one of the presenters, Dr. Gabriella Pridjian from Tulane Hayward Genetics Center, reported on another pregnant PKU patient who had difficulty tolerating low-phe protein supplements. She was instructed to divide the Sapropterin up twice daily and ultimately had a healthy baby delivered through c-section. Pridjian noted that there were plans to eventually formally test the baby (who was now three years old) for any intellectual or developmental issues.

In addition to these two case studies, the NIH panel reported that other pregnancies involving the use of Sapropterin were currently underway. In addition to concerns about whether Sapropterin harms a developing fetus, doctors admitted it was hard to determine if symptoms such as headache, rhinorrhea, vomiting, fever, abdominal pain, rashes or nasal congestion were a result of the prescription drug or simply associated with the pregnancy.

Even though the maker of Kuvan, BioMarin, does in fact encourage pregnant women who were exposed to Kuvan during their pregnancy to participate in ongoing studies about the drug’s effect, there are no plans for future controlled studies. Based on what little has been conducted, including the two case studies I’ve mentioned here, initial reports of using Sapropterin during pregnancy are encouraging. However, continued research on this matter is definitely a must, especially with concern to the long-term implications the drug might have on a PKU woman’s child.



Filed under Conferences & Events, Kuvan, Maternal PKU, Research

Starting my Kuvan Responder Trial

Kuvan LogoUp until four years ago, the only method for treating people with Phenylketonuria (PKU) was the low-protein diet. I remember being a young girl and my mother telling me that the second there was a cure, she would immediately sign me up. Nowadays, we still don’t have a cure, but we do have the first prescription drug for the treatment of PKU. It’s called Kuvan and it was developed by a pharmaceutical company called BioMarin. Here’s a brief video clip from the U.S. Food and Drug Administration announcing its approval.

Unfortunately, Kuvan doesn’t work for everyone, so before you can receive a prescription, you are required to go through trial to see if you actually respond to the medication. I will be starting my Kuvan trial this week and here’s how it breaks down:

PKU Kuvan Responder Trial

  • I should be receiving my package of Kuvan on Monday (that’s tomorrow)
  • Then on Thursday, I’ll start keeping a very specific food diary (notepad icon). I’m not yet on the low-protein diet, but my clinic advised that as long as I consume close to the same amount of protein each day then we should be in good shape. Otherwise, it would be impossible to tell if Kuvan was working or if I just ate less protein.
  • In addition to the food diary, I also have to take 7 blood samples (red circles). The blood work is pretty intensive in the beginning, but that’s to make sure we get a good baseline reading.
  • Then about three days into the trial, I’ll start taking my Kuvan (yellow circles). The number of pills you take depends on your body weight. I was prescribed to take 13 pills. I need to take Kuvan at about the same time every day and with food since it is acidic and can cause stomach problems.

At the end of the trial, we should know whether I have responded to Kuvan or not. I have some concerns about possible side effects, some of which can be very serious, and I haven’t made any final decisions whether I will take Kuvan even if I am a responder. I am confident that I will not take Kuvan during any pregnancies. Nonetheless, the more information I have, then the better my decisions will be.

Stay tuned and I’ll let you know how it goes!



Filed under Kuvan