Category Archives: Paediatrics

Study using DFMO shows positive results for children with high risk neuroblastoma

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A paper published September 27 in Scientific Reports shows the positive results of a phase II clinical trial using the oral medication DFMO to prevent relapse in children with High Risk Neuroblastoma (HRNB).

Neuroblastoma is a form of cancer that develops from immature nerve cells found in several areas of the body. It occurs most often in infants and young children, usually under the age of five. The disease remains a challenge in pediatric oncology and current treatments include therapies that have significant long-term side effects for patients.

HRNB accounts for 15 percent of all childhood cancer deaths, in part, due to the fact that nearly half of all patients who reach remission will relapse.

“These results are promising and have changed the outlook for our patients with high risk neuroblastoma,” said Giselle Sholler, MD, director of pediatric oncology research at Spectrum Health Helen DeVos Children’s Hospital and principal investigator of the study.

“By using DFMO for two years after finishing conventional therapy, we’ve seen an overall two-year survival rate for these children of 97 percent. This is a large increase in survival,” Sholler added. “Previously it was believed that children with refractory and relapsed neuroblastoma were considered incurable. This study shows more than 50 percent of patients remaining in remission up to four years.”

Beat Childhood Cancer’s trial studied the use of difluoromethylornithine (DFMO) as a single agent for enrolled patients at 20 children’s hospitals from June 2012 to February 2016. The children received two years of oral DFMO twice daily and were evaluated for outcomes of event free survival (EFS*) and overall survival (OS). The study used targeted oral therapy of an ODC inhibitor (DFMO), as a maintenance therapy to prevent relapse in HRNB patients after standard therapy. DFMO works by targeting specific cancer stem cell pathways and “turning off” the cells, thereby preventing the cancer from growing back.

There were two arms in this study, the first designed for patients who had completed standard therapy, and the second for children who were able to achieve remission after having previously relapsed. Both of these patient populations are at very high risk of relapsing after completing treatment and therefore can be very good candidates for using a maintenance therapy with the goal of preventing relapse.

With a median follow up of 3.5 years, the first arm of the study had 100 eligible patients. The results show that two-year EFS was 84 percent and two-year OS was 97 percent.

With a median follow up of 3.7 years, the study enrolled 39 previously relapsed patients and the results reported in the journal showed that two year EFS was 54 percent and two-year OS was 84 percent for these children who had previously relapsed.

“While these EFS and OS figures at two years are remarkable, the really exciting part of these results is that EFS and OS are stable out to four years,” said Patrick Lacey of Beat NB Cancer Foundation, one of the childhood cancer parent-led foundations that funded this clinical trial. “Not only did this oral drug lead to a prolonged and stable remission for the children in this study, but the drug was extremely safe and well tolerated in this patient population.”

“While many children have been able to attain remission with the current, albeit harsh, upfront therapies, these remissions are not historically durable,” Dr. Sholler added. “The current five-year survival curves have not changed significantly in the past two decades despite recent increases in two-year survival as a result of intensified therapies and new multimodal therapies.”

Principal Investigator at MUSC, Jaqueline Kraveka, MD, states survival for children with high-risk neuroblastoma remains a challenge. “These results are groundbreaking and very exciting for oncologists and their patient families. I am thrilled to have our confirmatory study open at so many sites across the USA and Canada, enabling children to receive this treatment close to home.”

SOURCE: www.news-medical.net/news/20181001

Urine exRNA may be source of biomarkers for muscular dystrophy

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Massachusetts General Hospital (MGH) researchers have found that extracellular RNA (exRNA) in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

The report published in online journal Nature Communications, is the first to show that urine exRNA can be used to monitor systemic diseases that do not directly affect the urinary tract.

“Our findings could facilitate drug development by offering a convenient, painless and relatively low-cost assay that may reduce and perhaps eventually eliminate the need for multiple invasive muscle biopsies to track disease activity and therapeutic response,” says Thurman Wheeler, MD, MGH Department of Neurology, senior author of the report. “Urine exRNA monitoring could determine whether a drug is reaching its target long before clinical effects on muscle function could be detected and may enable early identification of whether dosage adjustments may be required, something that would be impossible with invasive muscle biopsies.”

There are several types of muscular dystrophy, all of which lead to progressive muscle weakness and loss of muscle mass. Duchenne muscular dystrophy (DMD) is the most common form, with symptoms that usually begin in children under the age of 5. Myotonic muscular dystrophy (DM) is the most common adult-onset form and has two subtypes – DM1 and DM2. Each form is caused by a different gene mutation. In DMD, the mutation affects the gene for dystrophin, a protein essential to the strength of muscle fibers. DM-associated mutations – in the DMPK gene for DM1 and in the CNBP gene for DM2 – involve excessive repeats of nucleotides, leading to abnormal processing of RNA molecules.

The mutation associated with DM1 affects RNA splicing, the process that removes non-coding segments from an RNA molecule. A single gene can normally give rise to several different proteins, with the differences being determined by alternative RNA splicing patterns. The DM1 mutation interferes with appropriate splicing of RNAs encoding several other proteins, and analysis of RNA splice variants in muscle biopsies has been used to determine disease severity in patients. In animal models, splice variants in muscle tissue have been used to indicate whether potential therapies are reaching their molecular target. A less invasive way of assessing disease severity and therapeutic response could expand the number of patients who could receive therapeutic drugs or participate in clinical trials. For example, a recent clinical trial for a DM1 drug was restricted to adult patients partially because of the need for repeat muscle biopsies.

Carried through bodily fluids like blood and urine in membrane bubbles called vesicles, exRNA encompasses messenger, non-coding, and microRNA molecules and can reflect mutations, deletions and other molecular variants. A few muscular-dystrophy-associated RNA or protein biomarkers have been identified in the blood of patients. Even though it seemed unlikely that exRNA from the skeletal and cardiac muscle tissues affected by DM1 could pass through the kidney’s filtration system into the urine, urine is such an easily accessible fluid that Wheeler’s team analyzed vesicles from both blood and urine for exRNAs that could reflect results of the DM1 mutation.

Their experiments comparing urine exRNAs from DM1 patients, patients with two other forms of muscular dystrophy and unaffected control volunteers identified 10 transcripts that are alternatively spliced in a pattern unique to DM1 patients, most of which had been previously found in patient muscle biopsies. A composite biomarker incorporating these 10 transcripts was 100 percent accurate in distinguishing DM1 patients from unaffected controls in a different group of participants. Samples taken from untreated DM1 patients over several months indicated consistency of splicing patterns within an individual, suggesting that repeat sampling could accurately reflect disease state and treatment response.

Along with developing a more precise assay for rapid measurement of alternative RNA splicing in urine and other bodily fluids, the team showed that splicing patterns in total RNA from the cells in the urine were different from and less useful as biomarkers than those from exRNAs and found that exRNAs in blood could not distinguish between DM1 patients and controls. They also found that the splicing patterns of some urine exRNA transcripts reflected the severity of DM1 symptoms, and that a small group of asymptomatic patients with the DM1 mutation had urine exRNA splicing patterns midway between those of symptomatic patients and unaffected controls.

A group of drugs being evaluated for the treatment of DMD manipulate splicing of the dystrophin gene in order to remove a specific exon – a protein-coding segment of RNA – producing a shortened but partially functional version of the dystrophin protein. The MGH team showed that urine exRNAs from six untreated DMD patients accurately reflected the specific gene mutation in each patient. In two DMD patients being treated with eteplirsen – an FDA-approved DMD drug that induces skipping of the target exon – analysis of urine exRNA was able to confirm the drug was reaching its molecular target, the first such confirmation not provided by muscle biopsy.

“Our demonstration of disease-specific splice variants in urine exRNA suggests the value of biofluids as a means of identifying novel splice variants that may help correlate gene variants with symptoms for several diseases for which noninvasive biomarkers are unavailable,” says Wheeler, an assistant professor of Neurology at Harvard Medical School. “These findings support studies of exRNA from urine, blood or cerebrospinal fluid as biomarker replacements for tissue biopsies in other conditions with altered RNA splicing – including other types of muscular dystrophy, spinal muscular atrophy and amyotrophic lateral sclerosis.”

SOURCE: www.news-medical.net/news

Paracetamol use in infancy may increase the risk of asthma

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A study has found that paracetamol use in infancy, as well as genetic factors, may increase the likelihood of developing asthma.

A study by researchers from the University of Melbourne, Australia identified how infants who take paracetamol during the first two years of life could be at a higher risk of developing asthma in their late teens.

Xin Dai, a PhD candidate at the University, and her colleagues explained how the link between paracetamol use and asthma seemed to be the strongest in individuals with a particular variant of the glutathione S-transferase (GST) gene, GSTP1.

GSTP1 is part of a gene family which encodes gene crucial for some life processes, detoxification and toxification mechanisms. The genes are upregulated in response to oxidative stress, and are overexpressed in tumours.

GST genes contain the information needed to make the antioxidant glutathione, which clears the effect of exposure to toxins in the body and the lungs.These actions help to prevent damage to cells and inflammation.

“Paracetamol, on the other hand, consumes glutathione, reducing the body’s capacity to deal with toxic exposure,” said Ms Dai. “We hypothesised that people who did not have full GST enzyme activity because of common genetic variations or deletions may be more susceptible to adverse effects on the lungs from paracetamol use.”

The researchers investigated 620 children who had been followed form birth to the age of 18, as part of the Melbourne Atopy Cohort Study. These children were selected before birth, as it was deemed that they had a high risk of developing an allergy-related disease. At least one family member (father, mother or sibling) had a self-reported allergic disease, such as asthma, eczema, hay fever or a severe food allergy.

After the birth of each child, a nurse rang the family every four weeks for the first 15 months, and then at 18 months and at two years old to ascertain the number of time the infant was given paracetamol. As the child reached the age of 18, a blood or saliva sample was given and tests were conducted to identify variants of GST genes.

The participants were also assessed for asthma, and a spirometry test measured the amount of air inhaled and exhaled when breathing through a mouthpiece.

“We found that children with the GSTP1 Ile/Ile variant had 1.8 times higher risk of developing asthma by the age of 18 years for each doubling of the days of paracetamol exposure when compared to children who were less exposed,” said Ms Dai. “In contrast, increasing paracetamol exposure in children who had other types of GSTP1 did not alter the risk of asthma.

In addition, we found some weak evidence that paracetamol use in the first two years of life may be associated with reduced lung function in adolescence regardless of which variants of the GST genes the children had.”

The researchers stressed that the study showed an association between paracetamol and asthma, and not that one caused the other. To establish this, further research would be necessary.

She concluded: “Our findings provide more evidence that paracetamol use in infancy may have an adverse effect on respiratory health for children with particular genetic profiles and could be a possible cause of asthma. However, these findings would need to be confirmed by other studies and the degree of adverse effect better understood before this evidence could be used to influence practice and before guidelines on paracetamol use are altered.

“There is mounting evidence that the GST superfamily of genes, including three major classes -GSTM1, GSTT1 and GSTP1 – are associated with various diseases, including cancers, asthma, atherosclerosis, allergies, Alzheimer’s and Parkinson’s disease. Our study adds to this body of evidence.”

The study was presented at the European Respiratory Society International Congress.

SOURCE: www.europeanpharmaceuticalreview.com/news/79189

NICE U-turn on Crystiva for rare bone disease

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Reversing its initial decision to reject the drug, NICE has issued a positive recommendation for Kyowa Kirin’s rare disease drug Crystiva, the first treatment approved to target the underlying pathophysiology of X-Linked Hypophosphataemia (XLH).

The U-turn is good news for the Japan company, which negotiated on the price of the treatment behind closed doors after the NICE’s  conclusion that the drug wasn’t cost effective.

the UK’s cost-effectiveness watchdog is now set to recommend the twice-monthly injection for the treatment of XLH in children and young people with growing bones, with final guidance expected in October.

Tom Stratford, CEO, Kyowa Kirin International said: It is a major development that NICE has recommended Crysvita for routine use among children and young people with XLH in England and Wales.

“This marks a step change in treatment for XLH, emphasised through the emotional testimonies provided by patient groups and clinicians following the first evaluation consultation.”

Characterised by bowed or bent legs, a short stature, bone pain and delayed walking, XLH is first seen in infants but can also affect adults.

It is caused by low levels of phosphate in the blood, resulting in life-long physical disabilities.

Until now, treating this disease has consisted of multiple daily doses of phosphate and vitamin D to counteract the effects of FGF23, a protein that when produced excessively, reduces renal phosphates in the blood.

Crysvita targets this pathway by blocking the activity of FGF23, restoring phosphate blood levels by reducing phosphate loss via the kidneys.

Commenting on NICE’s decision, Oliver Gardiner, Board Member at XLH UK, said: “This is important news for children and young adults with XLH who will now be able to benefit from Crysvita routinely on the NHS.

“Access to a treatment that tackles the underlying mechanism and has the potential to avoid or mitigate substantial physical and emotional challenges, will truly make a difference to the lives of patients and their families.”

Crysvita was already accessible to patients under the NHS via the UK’s early access programme, which will be extended to allow time for NHS England to implement NICE’s final guidance.

SOURCE: www.pmlive.com/pharma_news

Novartis’ CAR T therapy Kymriah to become available on the NHS

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Novartis’ Kymriah is set to become the first CAR T therapy to become available on the NHS after it was revealed that the cancer treatment will be offered to children and young adults up to the age of 25 years old with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse.

While both Gilead and Novartis’ CAR T therapy were awarded marketing authorisation in the European Union just last week, NICE were quick to reject Gilead’s CAR T therapy Yescarta on the grounds that it was too expensive.

However Novartis’ one time cancer treatment is now set to become available through the UK’s national healthcare system.

Mari Scheiffele, Novartis Oncology General Manager, UK & Ireland, said: “This decision to make our revolutionary CAR-T therapy, Kymriah (tisagenlecleucel) available so soon after being licensed is the result of our close collaboration with NHS England and NICE, with flexibility shown by all parties to ensure young patients can access this life-saving treatment as quickly as possible.”

The custom made treatment, which uses an individual’s own immune cells to combat cancer, has the potential to extend survival and significantly improve quality of life for children and young adults whose prognosis is poor.

However the cancer therapy comes with a high price tag, costing $475,000 in the United States. Meanwhile the list price for Gilead’s alternative Yescarta is just $373,000 in the US. Nevertheless the price that has been negotiated between Novartis and NICE will be kept confidential.

SOURCE: www.pharmafile.com/news/518554

Kids are taking more alternative medicines than ever, and pediatricians are worried

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Supplements such as melatonin are marketed to parents, but not tested by the FDA.

Children and teens are turning to alternative medicines in soaring numbers, and the trend has pediatricians worried.

Children’s use of herbal products and nutritional supplements nearly doubled between 2003 and 2014, a study published Monday by the Journal of the American Medical Association found. Researchers at the University of Illinois at Chicago analyzed national health surveys of more than 4,400 young people and found that 6.7% took alternative medicines in 2014, compared to 3.7% in 2003. Some 33.2% of children and teens took a dietary supplement of some kind in 2014, including multivitamins.

Though they’re marketed as cures for a range of ailments, alternative supplements and medicines often end up on store shelves without oversight or approval from the federal Food and Drug Administration, the researchers noted. The surge in use was driven by 13 to 18-year-olds using omega 3 fatty acids — which are often sold as a way to boost mental focus — and melatonin, which is marketed as a sleep aid for kids, researchers found.

“We simply do not know if there are any benefits to children that outweigh the potential harms, and this study suggests supplement use is widespread and therefore an important, yet often ignored, public health issue,” said study co-author Dima Qato, assistant professor of pharmacy systems, outcomes and policy at the UIC College of Pharmacy.

“Many dietary supplements have also been implicated in adverse drug events, especially cardiovascular, which is a safety concern,” Qato said.

The findings come as America’s $3 trillion “wellness” industry is booming as consumers seek out alternatives to traditional Western medicine.

SOURCE: www.marketwatch.com/story

Roche SMA drug shines in study as costly new therapies advance

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A drug being co-developed by Roche to treat spinal muscular atrophy (SMA) helped improve development scores in babies with the genetic disease, a study released on Monday showed, as the race heats up for therapies destined to be among the drug industry’s most expensive.

PTC Therapeutics, which struck a licensing deal with Roche in 2011 for its SMA program, said more than 90 percent of babies with severe Type 1 SMA given the RG7916 drug achieved a greater than four-point increase in a test to measure their neuromuscular progress six months after treatment began.

PTC shares, which are listed on the Nasdaq, rose as much as 30 percent.

The companies hope their medicine, also known as risdiplam, will be approved to take on rival drug Spinraza from Biogen, which sells for $750,000 for the first year of therapy and about $375,000 annually after that.

Novartis is also quickly advancing in the SMA field with its $8.7 billion acquisition this year of U.S.-based Avexis that is working on a gene therapy for the disorder.

Analysts from Barclays project Novartis’s one-time treatment could run to $1.25 million per patient.

“We are delighted that up to 6.5-fold increase of protein production has translated into clinical impact for these babies,” PTC Chief Executive Stuart Peltz said in a statement about the RG7916 study, adding no babies required a tracheostomy or permanent ventilation since the study began, and no baby lost his or her ability to swallow.

SMA is an often-deadly genetic neuromuscular disorder caused by a missing or defective gene that normally produces a protein needed for development of motor neurons in the spinal cord. This leads to muscle wasting. Many babies with the severest form of the disorder die, while others never stand or walk.

Barclays analyst Emmanuel Papadakis expects that if Roche and PTC’s data holds up on RG7916 with regulators, it could become a fierce competitor to Spinraza, now the only licensed SMA therapy.

Cowen analysts also said on Monday they view PTC’s update as very encouraging for approval — and for competitiveness versus Spinraza, which is administered into the spine about four times a year. Roche’s and PTC’s drug is taken orally.

Novartis Chief Executive Vas Narasimhan also has high hopes for his own newly acquired SMA treatment, saying in April after the Swiss company bought Avexis that its gene therapy has “multi-billion dollar peak sales potential”.

SOURCE: www.pmlive.com/pharma_news

GSK’s blockbuster HIV drug linked with birth defects

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Regulators in Europe and the US have issued warnings about a link between GlaxoSmithKline’s blockbuster HIV drug Tivicay (dolutegravir) and birth defects of the brain and spine.

The European Medicines Agency’s safety committee has said it is evaluating preliminary results from a study which found four cases of birth defects such as spina bifida – a malformed spinal cord – in babies born to mothers who became pregnant while taking Tivicay (dolutegravir).

The Pharmacovigilance Risk Assessment Committee (PRAC) has issued precautionary advice while assessing the study, saying Tivicay should not be prescribed to women seeking to become pregnant.

Women who can become pregnant should use effective contraception while taking Tivicay-based medicines, the PRAC said.

In a separate announcement the FDA said it was also issuing a warning following preliminary results of an ongoing observational study in Botswana, suggesting women who took Tivicay at the time of becoming pregnant, or early in the first trimester, appear to be at higher risk for these birth defects.

The US regulator warned against stopping Tivicay-containing regiments without first switching to alternative HIV medicines, as this could cause the virus to spread to the unborn baby.

Women of childbearing age should talk to healthcare professionals about other non-Tivicay containing medicines.

Tivicay, an integrase strand transfer inhibitor (INSTI) is an important drug for GlaxoSmithKline, which produces the drug through its ViiV joint HIV venture with Pfizer and Shionogi.

Tivicay (dolutegravir) is sold on its own for use as a component of combinations involving other drugs, and generated sales of £1.4 billion,

And Triumeq, which combines dolutegravir with nucleoside reverse transcriptase inhibitors abacavir and lamivudine, brings in sales of more than £2.4 billion.

There are alternatives from the same class such as Merck & Co’s Isentress (raltegravir) and combinations, and combinations containing Gilead’s elvitegravir.

These are still preliminary findings, and the warnings have been issued purely as a precaution.

But preventing HIV spreading to unborn children is important to the medical community, and if there is stronger evidence of a link with birth defects, doctors will surely look for alternative therapies.

SOURCE: www.pharmaphorum.com/news

Oral antibiotics may raise risk of kidney stones

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Paediatric researchers have found that children and adults treated with some oral antibiotics have a significantly higher risk of developing kidney stones.

This is the first time that these medicines have been linked to this condition. The strongest risks appeared at younger ages and among patients most recently exposed to antibiotics.

“The overall prevalence of kidney stones has risen by 70 percent over the past 30 years, with particularly sharp increases among adolescents and young women,” said study leader Dr Gregory E. Tasian, a paediatric urologist at Children’s Hospital of Philadelphia (CHOP). Dr Tasian noted that kidney stones were previously rare in children.

Study co-author Dr Michelle Denburg, a paediatric nephrologist at CHOP, added, “The reasons for the increase are unknown, but our findings suggest that oral antibiotics play a role, especially given that children are prescribed antibiotics at higher rates than adults.”

The study team drew on electronic health records from the United Kingdom, covering 13 million adults and children seen by general practitioners in the Health Improvement Network between 1994 and 2015. The team analysed prior antibiotic exposure for nearly 26,000 patients with kidney stones, compared to nearly 260,000 control subjects.

They found that five classes of oral antibiotics were associated with a diagnosis of kidney stone disease. The five classes were oral sulfas, cephalosporins, fluoroquinolones, nitrofurantoin, and broad-spectrum penicillins. After adjustments for age, sex, race, urinary tract infection, other medications and other medical conditions, patients who received sulfa drugs were more than twice as likely as those not exposed to antibiotics to have kidney stones; for broad-spectrum penicillins, the increased risk was 27 percent higher.

The strongest risks for kidney stones were in children and adolescents. The risk of kidney stones decreased over time but remained elevated several years after antibiotic use.

Scientists already knew that antibiotics alter the composition of the human microbiome–the community of microorganisms in the body. Disruptions in the intestinal and urinary microbiome have been linked to the occurrence of kidney stones, but no previous studies revealed an association between antibiotic usage and stones.

Dr Tasian pointed out that other researchers have found that roughly 30 percent of antibiotics prescribed in office visits are inappropriate, and children receive more antibiotics than any other age group, so the new findings reinforce the need for clinicians to be careful in prescribing correct antibiotics. He added, “Our findings suggest that antibiotic prescription practices represent a modifiable risk factor–a change in prescribing patterns might decrease the current epidemic of kidney stones in children.”

One co-author of the current paper Dr Jeffrey Gerber, is an infectious diseases specialist at CHOP who leads programs in antibiotic stewardship–an approach that guides health care providers in prescribing the most appropriate antibiotic for each patient’s specific infection, with the aims of improving individual outcomes and reducing the overall risk of antibiotic resistance.

Dr Tasian and colleagues are continuing to investigate the microbiomes of children and adolescents with kidney stones in a single-centre study at CHOP. Their goal is to expand this research into broader, population-based studies to better understand how variations in microbiome composition may influence the development of kidney stones.

SOURCE: www.europeanpharmaceuticalreview.com/news/75652

FDA approves new drug to treat MS in pediatric patients

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The U.S. Food and Drug Administration today approved Gilenya (fingolimod) to treat relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older. This is the first FDA approval of a drug to treat MS in pediatric patients.

“For the first time, we have an FDA-approved treatment specifically for children and adolescents with multiple sclerosis,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “Multiple sclerosis can have a profound impact on a child’s life. This approval represents an important and needed advance in the care of pediatric patients with multiple sclerosis.”

Gilenya was first approved by the FDA in 2010 to treat adults with relapsing MS.

MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body. It is among the most common causes of neurological disability in young adults and occurs more frequently in women than men. For most people with MS, episodes of worsening function and appearance of new symptoms, called relapses or flare-ups, are initially followed by recovery periods (remissions). Over time, recovery may be incomplete, leading to progressive decline in function and increased disability. Most people with MS experience their first symptoms, like vision problems or muscle weakness, between the ages of 20 to 40. Two to five percent of people with MS have symptom onset before age 18 and estimates suggest that 8,000 to 10,000 children and adolescents in the U.S. have MS.

The clinical trial evaluating the effectiveness of Gilenya in treating pediatric patients with MS included 214 evaluated patients aged 10 to 17 and compared Gilenya to another MS drug, interferon beta-1a. In the study, 86 percent of patients receiving Gilenya remained relapse-free after 24 months of treatment, compared to 46 percent of those receiving interferon beta-1a.

The side effects of Gilenya in pediatric trial participants were similar to those seen in adults. The most common side effects were headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities.

Gilenya must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks. Serious risks include slowing of the heart rate, especially after the first dose. Gilenya may increase the risk of serious infections. Patients should be monitored for infection during treatment and for two months after discontinuation of treatment. A rare brain infection that usually leads to death or severe disability, called progressive multifocal leukoencephalopathy (PML) has been reported in patients being treated with Gilenya. PML cases usually occur in patients with weakened immune systems. Gilenya can cause vision problems. Gilenya may increase the risk for swelling and narrowing of the blood vessels in the brain (posterior reversible encephalopathy syndrome). Other serious risks include respiratory problems, liver injury, increased blood pressure and skin cancer. Gilenya can cause harm to a developing fetus; women of child-bearing age should be advised of the potential risk to the fetus and to use effective contraception.

The FDA granted Priority Review and Breakthrough Therapy designation for this indication.

The FDA granted the approval of Gilenya to Novartis.

SOURCE: www.news-medical.net/news/20180514