New Enzyme Replacement Therapy Announced in Malaysia to Treat Pompe Disease

WORDS LIM TECK CHOON

Sanofi Malaysia announced this month that Nexviazyme® (avalglucosidase alfa), a new enzyme replacement therapy (ERT), has been approved in Malaysia for the treatment of Pompe disease, a rare and progressive genetic disorder that is often fatal if left untreated.

A QUICK PRIMER ON POMPE DISEASE
  • It is caused by a deficiency in acid glucosidase (GAA).
  • This deficiency causes an accumulation of a substance called lysosomal glycogen in the body’s tissues. This primarily affects the muscles.
  • As a result, the affected person will experience symptoms like muscle weakness, breathing difficulties, and heart problems. They may require the use of wheelchairs and respiratory support.
  • Pompe disease can affect individuals of all ages, but it is broadly divided into infantile onset Pompe disease (IOPD) and late onset Pompe disease (LOPD).
BENEFITS OF NEXVIAZYME® 
  • It can improve the lung function and walking distance of people living with Pompe disease.
  • There is also evidence that it can also help improve their muscle strength.
  • Certain clinical studies indicated that people with infantile onset Pompe disease exhibited improved motor functions, despite showing suboptimal response to previous treatments.
  • Other clinical studies found that those with late onset Pompe disease exhibited improved function of their respiratory muscles and increased ability to walk farther.

Nexviazyme® is approved for use in long-term enzyme replacement therapy, delivered via a drip or intravenously every 2 weeks.

Reference: Sanofi Malaysia. (2024, May 15). New ERT Nexviazyme® (avalglucosidase alfa) MAL23086006AZ approved for treatment of Pompe disease [Press release, KKLIU 1420/EXP 2/11/24].

Hoping for a Baby? These Multivitamins May Increase Your Odds of Success!

WORDS LIM TECK CHOON

Data from a recent study conducted by Singaporean researchers suggests that multivitamin supplements that contain folic acid and iodine may be beneficial to women that are planning and hoping to conceive a child.

WHAT THE STUDY DID

The investigators interviewed 908 women between 18 and 45 years old that were trying to conceive. These women were enrolled in the Singapore PREconception Study of long-Term maternal and child Outcomes (S-PRESTO).

As an aside, S-PRESTO is a large-scale study on the effects of nutrition, lifestyle, and maternal mood prior to and during pregnancy on the effects of the baby’s genetic materials and the presence clinically important outcomes of the pregnancy. This study is beyond the scope of this article, though!

Back to this study, the investigators were interested in 2 things:

  1. Fecundity, or the measure of the chances of successful pregnancy
  2. Time to pregnancy, or the time between the first moment when the woman tries to conceive and the time of successful conception
WHAT THE INVESTIGATORS FOUND
  1. Multivitamins containing folic acid and iodine improve the success rate of having a baby when compared to those that do not take these supplements.
  2. These multivitamins also are linked to shorter time to pregnancy.
  3. Vitamins D and E do not show any significant improvements to pregnancy success rate and time to pregnancy.
  4. On the other hand, consumption of evening primrose oil is linked to lower pregnancy success rate and slightly longer time to pregnancy, compared to those that do not take this supplement.
WHY FOLIC ACID?

Folic acid, or vitamin B9, plays a role in breaking down and reducing the levels of an amino acid called homocysteine in the body.

High levels of homocysteine can cause effects that can affect fertility, such as increasing the ageing rate of a woman’s eggs and triggering the death of trophoblasts, which are cells of the outer layer of the developing embryo that helps it to attach to the woman’s endometrium for further development, protect the embryo, and eventually form the placenta.

Hence, by reducing the effects of high levels of homocysteine, folic acid can help improve the chances of a successful pregnancy.

HOW ABOUT IODINE?

Past tests on animals suggested that iodine can help improve the regulation of ovulation, increase the odds of a successful fertilization of an egg by a sperm cell following sexual intercourse, and promote successful implantation of the fertilized egg into the endometrium of the woman for further development.

Iodine also plays an important role in the development of nerve and brain cells of the foetus.

HOWEVER, SOME POINTS TO NOTE BEFORE YOU RUSH TO THE PHARMACY!

The investigators noted that more research is needed before we can make any conclusive statements about the effects of folic acid and iodine on one’s hopes of a successful pregnancy.

For example, their study did not measure the exact amounts of these nutrients that will work best on a woman hoping to conceive, and more research would be needed to determine whether their results can be replicated.

However, they believed that women trying to conceive could potentially benefit from taking multivitamin supplements containing these two nutrients, when this is also done in conjunction with other beneficial lifestyle modifications such as being physically active and cutting down on smoking and alcohol intake.

Reference: Ku, C. W., Ku, C. O., Tay, L. P. C., Xing, H. K., Cheung, Y. B., Godfrey, K. M., Colega, M. T., Teo, C., Tan, K. M. L., Chong, Y. S., Shek, L. P., Tan, K. H., Chan, S. Y., Lim, S. X., Chong, M. F., Yap, F., Chan, J. K. Y., & Loy, S. L. (2022). Dietary supplement intake and fecundability in a Singapore preconception cohort study. Nutrients, 14(23), 5110. https://doi.org/10.3390/nu14235110