Category Archives: FDA

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.


Eczema drug effective against severe asthma

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Two new studies of patients with difficult-to-control asthma show that the eczema drug dupilumab alleviates asthma symptoms and improves patients’ ability to breathe better than standard therapies.

Two new studies of patients with difficult-to-control asthma show that the eczema drug dupilumab alleviates asthma symptoms and improves patients’ ability to breathe better than standard therapies. Dupilumab, an injectable anti-inflammatory drug, was approved in 2017 by the Food and Drug Administration as a treatment for eczema, a chronic skin disease.

The more than 2,000 patients enrolled in the studies suffered from moderate to severe asthma. All used standard asthma inhalers, and some also took oral steroids to control their severe asthma symptoms.

In one study, the rate of asthma exacerbations was almost cut in half for those taking dupilumab compared with those taking a placebo. On average, patients taking a placebo had close to one exacerbation per day during the year of the study. Exacerbations are periods of sudden worsening of asthma symptoms such as wheezing, coughing, shortness of breath and tightness in the chest.

Although the drug significantly reduced asthma symptoms for all patients, dupilumab worked particularly well in patients with high numbers of a specific type of white blood cell, called eosinophils, circulating in the bloodstream. For those patients, asthma exacerbations were cut by two-thirds.

“This drug not only reduced severe symptoms of asthma, it improved the ability to breathe,” said Dr Mario Castro, the Alan A. and Edith L. Wolff Distinguished Professor of Pulmonology and Critical Care Medicine. “That’s important because these patients have a chronic disabling disease that worsens over time with the loss of lung function. So far, we do not have a drug for asthma that changes the course of the disease. Current drugs for severe asthma help reduce trips to the emergency room, for example, but they don’t improve lung function.”

The first study included about 1,900 patients of at least 12 years of age and with moderate to severe asthma requiring they use at least three different inhalers to control their symptoms. One inhaler contained a corticosteroid that reduces inflammation, another contained a long-acting bronchodilator that relaxes airway muscles, and the third was a “rescue” inhaler filled with albuterol, a short-acting bronchodilator that quickly opens up the airway in the event of a more severe asthma attack.

Patients taking these inhaled medications then were randomly assigned to receive either dupilumab or a placebo for one year. Patients receiving dupilumab — an injectable antibody — also were randomly assigned to a higher or lower dose of the drug. Neither patients nor their doctors knew whether they were receiving the drug or the placebo.

In addition to reduced symptoms, the patients receiving dupilumab showed improved lung function in a test of “forced expiratory volume.” This test measures the amount of air a person can force from the lungs during a deep exhale. Patients receiving dupilumab, regardless of dose, improved their lung function by approximately 130-200 millilitres greater than those receiving the placebo. In general, there were no significant differences between the patients receiving high and low doses of dupilumab.

Rates of emergency room (ER) visits and hospitalisations also were improved for patients receiving the drug. In the placebo group (with 638 patients), on average, 6.5 percent of the patients required an emergency room visit or hospitalisation due to asthma during the study. In the dupilumab group (with 1,264 patients), on average, 3.5 percent of patients needed emergency care or hospitalisation due to asthma.

Based on the second study, Dr Castro said another benefit of the drug could be the ability to wean severe asthma patients off of chronic oral steroids, which can cause debilitating long-term side effects, including stunted growth, diabetes, cataracts and osteoporosis. The second study included about 200 patients using the same inhaled asthma medications as patients in the larger trial, plus additional oral steroids — usually prednisone — to control their more severe symptoms. Half of the patients receiving dupilumab in this study were able to completely eliminate prednisone use. And 80 percent of dupilumab-treated patients were able to at least cut their doses in half. Patients on placebo also reduced prednisone use but to a lesser degree, likely because the protocols of participating in a clinical trial help asthma control generally.

Dr Castro said doctors would like to help patients rely less on steroids for asthma control because those with severe asthma can be forced to take these drugs for decades to enable them to breathe.

“I have patients who have had to stop working and go on disability because their asthma symptoms are so severe they can no longer function in the workplace,” Dr Castro said. “I’m excited about the potential of dupilumab because I have so many patients who have maxed out on available therapies and they still can’t breathe. It can become a very disabling disease.”

Patients receiving dupilumab did experience known side effects of the drug, including pain and swelling at the injection site and a short-term bump in the number of eosinophil cells in the blood. Five patients who received dupilumab and three patients who received placebo died during the study period. According to the investigators and descriptions of these patients’ medical histories, all suffered from multiple severe medical conditions, and none of the deaths was deemed related to the study protocol.

The studies will be published online in The New England Journal of Medicine.


Sun Pharma drug combo scores FDA approval in advanced castration-resistant prostate cancer

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Sun Pharma is celebrating with the announcement that its CYP17 inhibitor Yonsa (abiraterone acetate) has been awarded FDA approval in combination with methylprednisolone for the treatment of metastatic castration-resistant prostate cancer (mCRPC) in adult patients.

Yonsa has been designed using Churchill Pharmaceuticals’ SoluMatrix Fine Particle Technology manufacturing process to offer a micronised formulation of abiraterone acetate tablets, allowing it to be more efficiently absorbed in the body. The active ingredient is converted in vivo to abiraterone, an androgen biosynthesis inhibitor that inhibits 17 α-hydroxylase/C17,20-lyase (CYP17).

As part of an existing deal between the pair, Churchill Pharmaceuticals will receive upfront, commercial milestone payments and royalties related to sales of the drug in the US, where Churchill will handle marketing duties.

“We are pleased to add Yonsa to our growing oncology portfolio and continue to deliver on Sun Pharma’s commitment for enhanced patient access to innovative cancer therapies,” commented Sun Pharma’s North American CEO Abhay Gandhi.


AZ’s potassium drug Lokelma finally approved in US

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AstraZeneca badly needs new drugs on the market as several former blockbusters have been hit by generic competition – and finally its high potassium treatment Lokelma has been approved by US regulators.

The drug, a highly selective potassium-removing agent, has been approved at the third time of asking by the FDA, which had been concerned about issues at its manufacturing plant in Texas.

European regulators approved the drug formerly known as ZS-9 in March after their concerns over the issues were resolved, and after two previous rejections the US regulator is also satisfied with the technical arrangements at the facility.

AZ gained rights to the drug after buying ZS Pharma in 2015 for $2.7 billion and is designed to treat hyperkalaemia, where high potassium levels threaten kidney and heart function.

Lokelma (sodium zirconium cyclosilicate) will compete with Vifor Pharma group member Relypsa’s rival Veltassa (patiromer), which has been on the market for a few years in the US and Europe.

The Anglo-Swedish pharma has predicted sales in excess of $1 billion annually for ZS-9, although some analysts say this is a conservative estimate.

The risk of hyperkalaemia increases significantly for patients with chronic kidney disease (CKD) and for those who take common medications for heart failure (HF), such as renin-angiotensin-aldosterone system (RAAS) inhibitors, which can increase potassium in the blood.

To help prevent the recurrence of hyperkalaemia, RAAS-inhibitor therapy is often modified or discontinued, which can compromise cardio-renal outcomes and increase the risk of death.

Sean Bohen, chief medical officer at AstraZeneca, said: “The consequences of hyperkalaemia can be very serious and it’s reassuring for treating physicians that Lokelma has demonstrated lowering of potassium levels in patients with chronic kidney disease, heart failure, diabetes and those taking RAAS inhibitors.”

AZ badly needs the new sales – sales of its Crestor (rosuvastatin), a former blockbuster were down 38% in Q1, to $338 million, and overall revenues fell 4% to just under $5.2 billion.

The company is selling off its old and unwanted drugs to prop up revenues and reduce costs – but this can only be seen as a short-term measure before new revenues come on stream.

CEO Pascal Soriot also faces a shareholder revolt, after more than 37% of shareholders voted against or abstained at the firm’s annual meeting when asked to approve a £9.4m pay package for Soriot, down from £14.3 million last year.

Soriot has set a sales target of above $40 billion by 2023, despite the struggles getting new drugs to the market.


FDA greenlights first non-opioid therapy to manage symptoms of opioid withdrawal

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As the opioid epidemic continues to grip the US as the leading cause of deaths in those under the age of 50, the FDA has made an important leap with the approval of Lucemyra (lofexidine hydrochloride) for managing the symptoms of opioid withdrawal in adult patients – the first non-opioid therapy for this purpose.

While this is an important step forward, the therapy is not a cure-all solution and is not guaranteed to prevent withdrawal symptoms and may only lessen their severity, and is only approved for use for up to 14 days. These symptoms can include anxiety, muscle aches, agitation, sweating, nausea, vomiting, problems sleeping and drug cravings, which can occur both in patients with opioid use disorder and in those using the drugs as directed by a medical professionals.

An oral, selective alpha 2-adrenergic receptor agonist, Lucemyra works by reducing the release of norepinephrine, which is thought to be a key element in the symptoms of opioid withdrawal. The regulator’s decision was based on two trials of 866 patients who met the Diagnostic and Statistical Manual-IV criteria for opioid dependence who were physically dependent on opioids and undergoing abrupt opioid discontinuation. In these trials, it was found that patients taking Lucemyra reported lower severity of withdrawal symptoms according to the Short Opiate Withdrawal Scale of Gossop.

The fast-tracked decision comes off the back of a positive opinion in March from an independent FDA advisory committee. The regulator also specified that it requires 15 postmarketing studies to be conducted to support the drug’s longer-term use.

“As part of our commitment to support patients struggling with addiction, we’re dedicated to encouraging innovative approaches to help mitigate the physiological challenges presented when patients discontinue opioids,” said FDA Commissioner Scott Gottlieb. “We’re developing new guidance to help accelerate the development of better treatments, including those that help manage opioid withdrawal symptoms. We know that the physical symptoms of opioid withdrawal can be one of the biggest barriers for patients seeking help and ultimately overcoming addiction. The fear of experiencing withdrawal symptoms often prevents those suffering from opioid addiction from seeking help. And those who seek assistance may relapse due to continued withdrawal symptoms. The FDA will continue to encourage the innovation and development of therapies to help those suffering from opioid addiction transition to lives of sobriety, as well as address the unfortunate stigma that’s sometimes associated with the use of medication-assisted treatments.”


In Europe, Mylan’s rivals try to plug EpiPen shortages

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LONDON (Reuters) – European makers of emergency allergy treatments are stepping up production of alternative life-saving adrenaline shots to try to fill intermittent shortages of Mylan’s (MYL.O) market-leading EpiPen injection.

Mylan began warning about EpiPen supply constraints in Britain two months ago. Canada has also seen similar problems, while on Wednesday the Food and Drug Administration added EpiPens to its list of drugs in shortage in the United States.

The shortfall reflects manufacturing delays at Pfizer’s (PFE.N) Meridian Medical Technologies unit, which is Mylan’s manufacturing partner and produces all the EpiPens sold globally at a single plant near St. Louis.

Allergy charities said there were anecdotal reports of some patients having difficulty filling prescriptions but there did not appear to be major supply issues overall, thanks to the availability of rival products.

Jext and Emerade, from ALK-Abello (ALKb.CO) and Valeant’s (VRX.TO) Bausch+Lomb unit respectively, are sold in both Britain and parts of Europe, while Lincoln Medical makes Anapen for certain European markets outside the UK.

“ALK has increased its production,” a spokesman for Denmark-based ALK-Abello said on Thursday. “We are doing all we can to meet the increased demand. We can make up some of the shortfall but not all, as EpiPen has a market share of around 70 percent.”

Lincoln Medical said it had not yet seen any major impact in Europe, reflecting the fact that the market was cushioned by multiple sources of supply and by the stocks held at distributors.

A spokeswoman for Britain’s health department said “limited” supplies of standard-dose EpiPens were available and stocks were being closely managed to ensure pharmacies could fulfil prescriptions. Supplies of half-dose 0.15 mg EpiPen junior have not been hit and remain readily available.

“Any patient who is unable to obtain supplies of EpiPen 0.3 mg should speak to their doctor about using an alternative,” she said.

EpiPens and other competing devices deliver doses of adrenaline via an automatic injector that a patient or caregiver can administer in the event of severe allergic reaction, such as to bee stings or exposure to peanuts.


FDA grants fast track designation for a Chikungunya vaccine

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Fast Track program is designed to facilitate development and expedite review of therapies and vaccines to address unmet health needs.

PaxVax has announced that it has received Fast Track designation from the US Food and Drug Administration (FDA) for its vaccine for the prevention of disease caused by the chikungunya virus. Chikungunya represents a significant public health need and can cause a headache, muscle pain, and skin rashes, with severe, often debilitating, joint pain that can persist for years, especially in adults.

This Fast Track designation is another positive step for the development of our chikungunya vaccine

The virus spreads through mosquito bites and can often cause large outbreaks. A recent study by the US Centers for Disease Control and Prevention (CDC) indicates that the number of people getting infected with diseases transmitted by a tick, flea and mosquito bites, such as chikungunya, has more than tripled in the U.S. in recent years.

“This Fast Track designation is another positive step for the development of our chikungunya vaccine, in addition to our recent Phase 2b initial patient enrollment, and is a clear recognition of the serious unmet need,” said Dr Lisa Danzig, Chief Medical Officer of PaxVax. “This is another example of the capabilities of our technology platform and our steadfast commitment to developing speciality vaccines that protect against overlooked infectious diseases as mosquito-borne diseases continue to increase due to air travel and warmer weather.”

The vaccine was licensed from the National Institute of Allergy and Infectious Diseases (NIAID) at National Institutes of Health (NIH). PaxVax recently announced the enrollment of the first patient in its Phase 2b dose-finding trial of the chikungunya virus-like particle (VLP) vaccine, building upon a Phase 2a study by the NIH with 400 subjects. The Phase 2b study is in the process of enrolling 400 subjects to evaluate multiple dosing regimens. PaxVax expects the results in early 2019.

The chikungunya virus has caused millions of infections worldwide in the past few years. In late 2013, the first local transmission in the Americas was identified in the Caribbean. Since then, chikungunya has been identified in 45 countries in the Americas alone with more than 1.7 million suspected cases reported to the Pan American Health Organization, increasing the incidence of the disease and risk to US travellers. In 2016 and 2017, cases were reported in the United StatesItaly and France. Additionally, in 2016 there were approximately 60,000 cases of chikungunya across India, posing a large burden on the healthcare system. Beyond the Indian subcontinent, the CaribbeanCentral America and South America, inhabitants and travellers visiting sub-Saharan Africa and Southeast Asia are also at risk.

“It is exciting to see this vaccine candidate moving forward with Fast Track designation as this is an extremely debilitating disease with the potential for causing serious long-term sequelae,” said Dr Eva Harris, from the Division of Infectious Diseases and Vaccinology and Director, Center for Global Public Health, University of California, Berkeley. “As there are currently no therapies or vaccines available for treatment or prevention of chikungunya, we are in desperate need for a medical and public health intervention.”

VLP vaccines are multi-protein structures that mimic the organisation and conformation of naturally occurring viruse, but lack the viral genome. They are non-infectious. Additionally, the chikungunya VLP vaccine maintains natural epitopes to mimic natural infection.

About Chikungunya

Chikungunya virus is an arthropod-borne virus (arbovirus), closely related to other viruses in AfricaSouth America and Australia that cause similar symptoms. Chikungunya virus is a small, spherical RNA virus and a member of the Alphavirus genus in the family Togaviridae. The virus is vectored by the daytime-biting Aedes aegypti mosquito, which also transmits yellow fever, Zika and dengue viruses. The virus originated in sub-Saharan Africa and has variants that spread throughout tropical areas of the world.

Chikungunya can also be transmitted by Aedes albopictus mosquitoes, a more cold-tolerant mosquito – this could result in the spread of chikungunya to more temperate areas of the world. Individuals who are at higher risk for more serious complications include infants, the elderly and those with chronic medical conditions. There are currently no FDA approved vaccines to prevent chikungunya and no specific treatments for the complications of the infection. Non-vaccine interventions to prevent infection are limited to using insect repellent, wearing long sleeves and pants and otherwise restricting exposure to vector mosquitos.


Sanofi’s latecomer PD-1 gets date for FDA verdict

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While Keytruda extends its lead, Sanofi makes a late bid to enter PD-1 space.

The FDA has set a deadline of 28 October for its review of Sanofi’s cemiplimab for a form of skin cancer, as the company tries to join the fast-growing PD-1/PD-L1 inhibitor market.

The US regulator has given a priority review for PD-1-targeting cemiplimab because its first indication – cutaneous squamous cell carcinoma (CSCC) – is the second most common form of skin cancer after basal cell carcinoma (BCC) and the deadliest after melanoma, with no approved therapies.

Regeneron-partnered cemiplimab is the first of the new wave of checkpoint inhibitors to be filed for CSCC, and also picked up a breakthrough designation from the FDA last September. It was filed in Europe in April, setting up a potential approval in early 2019.

With five drugs targeting PD-1 or PD-L1 already on the market, Sanofi and Regeneron are late entrants into the category. Selecting CSCC for the first marketing application will however allow them to build a niche for cemiplimab before going head-to-head with other drugs such as Bristol-Myers Squibb’s Opdivo (nivolumab) and Merck & Co’s Keytruda (pembrolizumab).

That said, it may not have the market to itself for very long. Keytruda is in phase II testing for CSCC, and the wealth of clinical experience with the drug will make it a potent rival even if Sanofi and Regeneron have first-mover advantage.

The drug is one of a new crop that Sanofi is counting on to stimulate renewed growth, after a difficult period caused by the loss of patent protection for diabetes blockbuster Lantus (insulin glargine) and a poor start for dengue vaccine Dengvaxia.

At its R&D day towards the end of 2017, the French pharma group took pains to highlight cemiplimab’s potential as a first-line therapy for non-small cell lung cancer (NSCLC), an indication which is fast becoming the main battleground for the PD-1/PD-L1 class and at the moment is dominated by Keytruda. Sanofi has three trials of cemiplimab either ongoing or planned in first-line NSCLC.

The company is pushing forward with its checkpoint inhibitor on a number of fronts, however, and is also expecting to report data in the coming months in BCC, another cancer for which none of the first five PD-1/PD-L1 drugs have been approved, as well as cervical cancer. It also has phase II trials ongoing in combination with Sanofi’s CD38-targeting antibody isatuximab in solid tumours and blood cancers, and early-stage studies of cemiplimab with DNA vaccines and oncolytic viruses.

Priority review for Keytruda in NSCLC

Meanwhile, Merck (known as MSD outside the US and Canada) had some good news of its own from the FDA this week. The agency has granted Keytruda a priority review in combination with Lilly’s Alimta (pemetrexed) and platinum-chemotherapy as a first-line treatment for patients with metastatic non-squamous NSCLC.

The filing is based on the KEYNOTE-189 study, which showed Keytruda improved overall survival in this patient group, and is due for judgment by the FDA on 23 September.


Allergan’s oral migraine drug closer to US filing

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Allergan has reported additional phase III results for its oral CGRP inhibitor ubrogepant, setting up a regulatory filing in the US next year.

New data keeps Allergan ahead of rival Biohaven

The positive read-out from its second pivotal trial for the acute treatment of a migraine attack showed a 50mg dose of ubrogepant was more likely to achieve a statistically significant reduction in pain two hours after administration – and another ‘most bothersome symptom’ (MBS), such as sensitivity to light or loud sounds or nausea – compared to placebo.

A lower 25mg dose also reduced pain but missed the mark on the MBS measure, according to Allergan, although it did show a trend towards improvement compared to the control group.

The company has reiterated that it is preparing to file the drug next year in the US, armed with the new data and an earlier study which showed significant efficacy across both endpoints for 50mg and 100m doses of the drug.

CGRP-targeting drugs look set to be the next wave of innovation in migraine treatment, with four companies vying for approval of intravenous or injectable candidates to be given either every month or every three months to try to prevent migraines occurring. Allergan’s drug is in the lead among a new group of orally-acting candidates – known collectively as ‘gepants’ – for acute use, in other words to try to provide relief of symptoms when an attack is already underway.

Novartis and Amgen are in pole position among the injectable CGRP-targeting drugs, with their Aimovig (erenumab) already filed and expected to gain FDA approval by May 17.

In Allergan’s new ACHIEVE II study, ubrogepant provided freedom from pain after two hours in 20.7% of the 25mg group and 21.8% of the 50mg group, compared to just over 14% of placebo-treated patients. The most bothersome symptom was relieved at that timepoint in 38.9% of the high dose group, 34.1% of low-dose patients and 27.4% of those on placebo.

The CGRP inhibitors offer hope to patients with severe chronic migraine who can’t get relief from the current array of therapies, which consists of pain-killing drugs, triptans and repurposed medicines such as the anticonvulsant topiramate or Allergan’s biggest and best-known product, Botox.

Analyst David Maris at Wells Fargo said Allergan’s new drug seems to offer triptan-like pain relief with an improved safety profile, and could be an option for an estimated four million people in the US with migraine who do not respond to or cannot tolerate triptan drugs, with blockbuster sales potential.

There were a few cases of raised aminotransferase levels (10 across the two studies at last count) – a potential market for liver toxicity – but according to Allergan are not judged to have been drug-related.

Ubrogepant’s nearest rival is Biohaven’s late-stage candidate rimegepant, which reported results from its phase III programme in March.

Allergan’s efforts to extend its migraine franchise suffered a setback when its inhaled drug Semprana (dihydroergotamine mesylate) was turned down by the FDA three times as an acute treatment for the disorder because of manufacturing issues. The programme is no longer listed in the company’s R&D pipeline.


ERT receives sixth consecutive award for outstanding customer service

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ERT, a global data and technology company that minimises uncertainty and risk in clinical trials, has received Omega Management Group Corporation’s NorthFace ScoreBoard (NFSB) Award for consistent delivery of outstanding customer service throughout 2017.

This marks the sixth consecutive year that ERT has been recognised for achieving excellence in customer satisfaction, as rated solely by its customers.

“The NorthFace ScoreBoard Award recognises organisations who, like ERT, not only offer exemplary customer service, but who also center their existence on a deep commitment to exceeding customer expectations,” said John Alexander Maraganis, President and CEO of Omega.

“There is no other award that recognises such achievement at this high level, so we are pleased to once again congratulate ERT for their ongoing efforts in this regard.”

Omega’s methodology measures customer satisfaction and loyalty levels on a five-point scale (or equivalent) four times during the year in such categories as technical support, field service, customer service and account management.

NFSB Award recipients are companies who, based solely on survey responses from their own customers, achieved a 4.0 or above out of a possible 5.0.

“Each of our employees is dedicated to helping our customers identify trial risks before they become problems to avoid costly delays,” said James Corrigan, President and CEO of ERT.

“They are the force behind our best-in-class solutions, services and practices that supported more than 60% of all FDA-approved drugs in 2017.”

“Although we are honoured that our customers continue to recognise ERT as a trusted partner, we are always looking ahead for new ways to help them mitigate risks and bring life-changing treatments to patients more quickly.”