GLOBAL NEWS MAY 16

 

Sovaldi/Olysio Combo Cures 94% of Those With Liver Disease

In a recent trial, combination therapy with Gilead Sciences’ Sovaldi (sofosbuvir) and Janssen’s Olysio (simeprevir) cured 94 percent of people who had genotype 1 of hepatitis C virus (HCV) and advanced liver disease, MedPage Today reports. Eagerly anticipated results from the Phase II COSMOS trial were presented at the 49th annual meeting of the European Association for the Study of the Liver (EASL) in London.

Eighty-seven study participants with F3 or F4 METAVIR fibrosis scores were divided into four groups: One took the NS5B nucleotide polymerase inhibitor Sovaldi plus the NS3/4A protease inhibitor Olysio for 12 weeks, and the other took the combination for 24 weeks; two other groups added ribavirin to the regimen and were treated for either 12 or 24 weeks.

Neither treatment length nor ribavirin use affected the success rate. In the 24-week groups, 93 percent (28/30) of those who took ribavirin achieved a sustained virologic response 12 weeks after completing therapy (SVR12, considered a cure), compared with 100 percent (16/16) who did not take ribavirin. In the 12-week groups, 93 percent (25/27) of those who took ribavirin were cured, compared with 93 percent (13/14) of those who did not take ribavirin.

Notably, those who had the Q80K polymorphism had the same outcomes as those who did not. Past research has found that the polymorphism lowers cure rates among those treated with protease inhibitors. Sovaldi apparently canceled out this effect.

While the U.S. Food and Drug Administration has not approved Sovaldi and Olysio for use together, many physicians are apparently prescribing them together off-label, using past research as a guide. Earlier in the year, the American Association for the Study of Liver Diseases recommended 24 weeks of Sovaldi, Olysio and ribavirin for people with genotype 1 of hep C who are interferon intolerant. 
 

http://www.hepmag.com/articles/Olysio_Sovaldi_2501_25501.shtml

 

The Hepatitis C Blockbuster Season Has Begun

 

by Benjamin Ryan

With the FDA approval of Sovaldi (sofosbuvir) and Olysio (simeprevir), many people with hepatitis C, including those coinfected with HIV, will likely line up for long-stalled treatment. Sovaldi in particular has the potential to reap billions in sales.

It’s here, and it’s expensive. 

On December 6, the U.S. Food and Drug Administration (FDA) approvedGilead Sciences’ hotly anticipated Sovaldi (sofosbuvir), granting a broad indication for its use to treat people with genotypes 1 through 4 of hepatitis C virus (HCV), including those coinfected with HIV and those who have liver cancer and are waiting for a transplant. 

“This is a very significant advance,” says Steven L. Flamm, MD, the chief of the liver transplant program at the Northwestern University Feinberg School of Medicine in Chicago, who has been conducting ongoing trials of Sovaldi in partnership with Gilead.

For the past two and a half years, the standard treatment for hep C has been a regimen of one out of two protease inhibitors—Incivek (boceprevir) and Victrelis (telaprevir)—paired with interferon and ribavirin. Now the nucleotide analog polymerase inhibitor Sovaldi promises to cut standard treatment time from the previous range of 24 to 48 weeks down to 12 to 24 weeks, in addition to lessening side effects and raising cure rates from about 70 percent to the 80 to 90 percent range in many cases.

Most groundbreakingly, Sovaldi is the backbone of the first-ever interferon-free, all-oral hep C drug regimen. While the once-daily pill must still be taken in combination with ribavirin, those with genotypes 2 and 3 of the virus can now avoid the weekly injection of interferon and its onerous, flu-like side effects. And to the surprise of many in the hep C community, the FDA has given clinicians the option of considering interferon-free treatment for people with genotype 1 who are interferon ineligible. This could provide the option of interferon-free treatment to vast swaths of Americans, because 70 percent of U.S. cases of hep C are genotype 1 and many physicians believe that simply not wanting to take interferon qualifies someone as ineligible for interferon. (Opting for this treatment route would be a gamble, however, because cutting interferon for treatment of genotype 1 also lowers the likelihood of a cure.)

The cost of this miracle drug? $1,000 a day.

Granted, Sovaldi’s price tag is still in the same general stratosphere as the therapies it will most likely eclipse, but physicians and advocacy groups such as the AIDS Healthcare Foundation and the Fair Pricing Coalition have expressed outrage at what they see as a brazen and greedy move on Gilead’s part. 

The pharmaceutical giant stands to reap a colossal reward for its investment in hep C therapeutics. Analysts with BMO Capital Markets expect Sovaldi’s 2014 revenue to come in at around $1.9 billion. Matthew Roden, an analyst at UBS, has said that the drug’s annual sales could eventually climb higher than the $13 billion brought in by Pfizer’s cholesterol-lowering Lipitor (atorvastatin) in its biggest year. 

Outside of how Sovaldi’s exorbitant cost may tax the U.S. health care system, the overriding concern among hep C advocates is over access to treatment for those without robust insurance coverage.

“I think it’s a potentially dangerous bar that will hopefully not be the floor for subsequent drugs that are going to be approved in the coming years,” Michael Ninburg, executive director of Hepatitis Education Project in Seattle, says of Sovaldi’s price tag.

Receiving the FDA nod two weeks before Sovaldi, Janssen Therapeutics’ NS3/4A protease inhibitor Olysio (simeprevir) costs 21 percent less: $66,360 for 12 weeks of treatment, compared with $84,000 for Sovaldi’s 12-week regimen. (Because 24 weeks of Sovaldi are recommended for treating genotype 3 and for treating genotype 1 without interferon, the cost would double in these cases.) Olysio is only approved for use among those with genotype 1 of the virus, with a recommended treatment regimen of 12 weeks combined with either 24 or 48 weeks of pegylated interferon and ribavirin, depending on past treatment experience. 

In pooled results from the Phase III QUEST-1 and QUEST-2 trials, Olysio’s sustained virologic response (SVR, considered a cure) rates ranged from a high of about 80 percent for treatment-naive study participants and for those who had relapsed on a previous treatment, down to 65 percent for prior partial-responders to treatment and 53 percent for prior null-responders. 

A major factor weighing against Olysio’s potential for widespread use is the fact that, among those with genotype 1a who have what’s known as a Q80K polymorphism, just 58 percent achieved a cure in Phase III trials. Nearly half of Americans with genotype 1a have the polymorphism.

Meanwhile, in the NEUTRINO Phase III trial, 12 weeks of Sovaldi in combination with interferon and ribavirin boasted a cure rate of 89 percent among genotype 1 participants—and that was without the need for the extended “tail” of the latter two drugs, as with Olysio. (Breaking the results down by subtype, the cure rate for genotype 1a was 92 percent and for 1b it was 82 percent.) In the PHOTON-1 study of an interferon-free 24-week regimen of Sovaldi and ribavirin, 76 percent of treatment-naive study participants with genotype 1 achieved an SVR.

Kris Kowdley, MD, a clinical professor of medicine at the University of Washington in Seattle, argues that Olysio’s lower cost is unlikely to play much in the drug’s favor, while both the Q80K polymorphism concern and Olysio’s longer period of treatment with interferon and ribavirin will weigh against the drug.

Meanwhile, pharmaceutical companies such as AbbVie, Bristol-Myers Squibb and Boehringer Ingelheim are all racing to get their own hep C drugs to market over the next two years, with a wave of advanced clinical trials researching combination therapies that have been pushing average SVR rates ever-closer to 100 percent. Gilead is also gunning for FDA approval, likely to come at the end of 2014, for a coformulated pill of Sovaldi along with the company’s investigatory NS5A inhibitor ledipasvir. Thus far, cure results in trials of the two drugs have been near perfect.

While the price of Incivek and Victrelis steadily rose after they were released, a cost war could lower prices of the new crop of drugs in the coming years. Express Scripts, the nation’s largest pharmacy benefits manager, has indicated that the company may exclude pricier hep C therapies from formularies down the road if these drug regimens offer no greater benefit in outcome over their cheaper competition. So even if Gilead’s once-a-day pill offered unique convenience, it still might get nixed for coverage in favor of a more cheaply priced multi-pill regimen from AbbVie, should the two companies’ regimens promise comparable cure rates.

As for now, both Sovaldi and Olysio are available and insurers are weighing their options. While this process can take three to six months, there is still the possibility of receiving coverage before the ultimate say-so is issued. Lorren Sandt, the Fair Pricing Coalition’s HCV co-chair, says she is particularly concerned about how the high cost of treatment may restrict access to those receiving medical coverage through Medicare and state Medicaid programs, AIDS Drug Assistance Programs (ADAP), the Veterans Administration and in correctional facilities. Another worry among some hep C advocates is that individual insurers may opt not to cover the newer treatments unless someone has already failed treatment with the older drugs. 

Sandt says that those concerned about access because of lack of insurance, high deductibles or even co-pays should take heart in the particularly generous patient assistance programs (PAPs) that Janssen and Gilead have set up to ease the sticker shock.

“I don’t want patients to see the price and think that they can’t afford it,” Sandt says. “Follow up with your doctor, find out if you’re a candidate for therapy. And if you are and you can’t afford it, make sure that you access the drug assistance programs.”

Gilead, for one, will provide financial assistance for Sovaldi to those who have no insurance and who have a maximum household income of $100,000 for a family of three and 500 percent of federal poverty level for larger households. There is assistance to bring co-pays down to no more than $5 in most cases, and financial aid for as much as $16,000 is available to go toward prescription deductibles and coinsurance obligations. Gilead is also partnering with an independent non-profit, the Patient Access Network (PAN) Foundation, which assists both federally- and privately-insured people who need assistance with out-of-pocket medication expenses. For more information on financial aid for Sovaldi, click here, and for Olysio, click here.

When it comes to deciding who should receive treatment now as opposed to waiting for greater developments in treatment, Northwestern’s Steven Flamm says elements to weigh include how advanced a patient’s liver disease may be and what kind of other symptoms, such as overwhelming fatigue, may add urgency to a particular case. Then there is simply the matter of highly motivated patients, those who are eager to rid themselves of the virus for any number of reasons and should be given the option to do so.

Considering Sovaldi treatment, the University of Washington’s Kris Kowdley says, “There’s no question that for genotype 2, there is more or less very little room to improve,” when Phase III trials of a 12-week regimen of the drug with ribavirin brought in SVR rates greater than 90 percent for those without cirrhosis.

For people with genotype 3, “especially in the commonly encountered genotype 3 patient that is treatment experienced and has cirrhosis,” Kowdley says that the prescribed 24 weeks of treatment with Sovaldi “is a very reasonable approach, given 60 percent SVR. But we still have opportunities to improve that further.” 

The results Kowdley refers to come from the Phase III VALENCE trial, in which cure rates ranged between 85 and 93 percent for treatment-naïve participants either with or without cirrhosis and for treatment-experienced participants without cirrhosis.

And for treatment-naive people with genotypes 1, 4, 5 and 6, Kowdley describes the 12-week regimen of Sovaldi, ribavirin and interferon as “very attractive,” considering its 90 percent SVR rate in the NEUTRINO Phase III trial. (Sovaldi is only approved for genotypes 1 through 4, but clinicians may still prescribe the drug off label to treat genotypes 5 and 6, using this trial as a guide.)

Reflecting on these same genotypes, Kowdley says, “I think there is clearly evidence from Phase II data from smaller studies that, in the absence of interferon, there may also be a role for [Sovaldi’s use in] treatment-experienced patients, and there may be a role for prior [protease inhibitor]-treated patients.”

Physicians may also opt to prescribe Sovaldi and Olysio together off label. Phase II research of the pair given for 12 weeks demonstrated 93 to 100 percent cure rates among genotype 1 null responders who had mild to moderate fibrosis.

“Now, whether insurance is going to cover this—when we say ‘that’s the million dollar question’ facetiously, that’s not that far from the truth,” Flamm says, “because it’s going to be very costly to use those two medications together. I think there will be a significant impetus for third-party payers to try to adhere to the approved regimen and not cover these drugs. But people are going to try.”
 

http://www.hepmag.com/articles/sovaldi_olysio_2502_24943.shtml



Gilead says has discounted hepatitis C drug for some health plans

By Deena Beasley

(Reuters) - Gilead Sciences Inc, under fire for pricing a new hepatitis C drug at $1,000 a pill, has discount agreements with a number of health insurers, a company executive said in an interview.

The medication, Sovaldi, has a list price of $84,000 for a 12-week course of therapy and is seen as a breakthrough in the treatment of the serious liver disease.

It has been shown to raise cure rates and cut treatment time with fewer side effects than older medicines, but critics maintain that a price of $1,000 each is too high for an easy-to-make pill needed by millions of Americans.

On March 20, Democratic lawmakers led by California Representative Henry Waxman asked Gilead to explain the price tag, and a meeting with the company is scheduled for next week.

Health insurers and state Medicaid programs for the poor are pushing for further discounts, fearing a multibillion-dollar price tag from treating most hepatitis C sufferers with Sovaldi and similar new medicines likely to be approved in coming years.

"It's the volume (of patients) payers are looking at here. It's not the price," said Gregg Alton, Gilead's executive vice president, corporate and medical affairs. "A lot of them are looking for a discount, but I think the real issue here is how many patients they now have in their plans that need hepatitis C treatment."

The Centers for Disease Control and Prevention estimates that about 3.2 million Americans are infected with hepatitis C, a liver-destroying virus transmitted through blood.

If each was treated with full-priced Sovaldi, the cost would be $269 billion. Gilead already provides a mandated discount off its list price to U.S. government health plans and insurers at about 23%.

Alton said the company has deals for "supplemental discounts" for government-funded agencies such the Veterans Administration and the Department of Defense, on top of the 23%. He would not provide details.

He said the VA, which accounts for about 10 to 15% of the hepatitis C population in the United States, has been "proactive" in recognizing the need to treat the disease, which can lead to liver failure and necessitate liver transplants.

He estimated that patients eligible for Medicaid, the government-funded health plan for the poor, account for another 10 to 15% of Americans with hepatitis C.

Alton singled out health maintenance organization Kaiser Permanente for taking action to secure Sovaldi, also at a discount, for its patients.

"We have an arrangement with Kaiser that works very well for both of us," the Gilead executive said. "They recognize that if they make the investment today, they get all the benefit."

He said that is because many Kaiser patients stay for many years with the organization known for quality, integrated care, even into retirement when they qualify for a managed Medicare plan. So Kaiser's upfront investment treating hepatitis C will pay off years later by averting future costs of liver disease. Kaiser officials were not immediately available for comment.

But many traditional insurers can't rely on the same kind of stability among their policyholders, who tend to switch health plans more frequently, meaning they cannot be sure of the same savings over time.

"One of the challenges we have with some insurers is that the benefits may not come to them," the Gilead executive said. "No matter how we price this product, the benefits and the savings are going to come later."

Alton also sees the concerns over a deluge of patients demanding immediate treatment as unlikely to materialize.

"Most of the patients are not diagnosed, and many aren't seeking care currently," he said. "This is going to take some time."

 
 http://www.reuters.com/article/2014/03/29

 

Nonalcoholic fatty liver disease linked to procoagulant imbalance

Last Updated: 2014-04-02 15:48:41 -0400 (Reuters Health)

By Will Boggs MD

NEW YORK (Reuters Health) - Patients with nonalcoholic fatty liver disease (NAFLD) have a procoagulant imbalance that worsens as the disease progresses, researchers from Italy report.

"Knowing that NAFLD is associated with an increased risk of cardiovascular disease and liver fibrosis, I thought that plasma hypercoagulability has something to do with NAFLD," Dr. Armando Tripodi from Universita degli Studi di Milano told Reuters Health by email.

He added that the new study, online March 19 in the Journal of Hepatology, is the first to demonstrate such hypercoagulability by means of a global coagulation assay.

Although a link between a hypercoagulable state and the increased cardiovascular risk in NAFLD had been proposed before, a direct link had not been established, perhaps because of the lack of appropriate laboratory tests for exploring coagulation under conditions mimicking those in vivo, the researchers write.

They used two thrombin-generation assays (endogenous thrombin potential (ETP) and Protac-induced coagulation inhibition (PICI)) to investigate the overall coagulant balance in 113 patients with NAFLD.

Dr. Tripodi noted that the assays are still research tools, but could become useful in the future for risk assessment in individual patients.

The researchers also used the ratio of factor VIII to protein C as their traditional index of the procoagulant imbalance (with higher values reflecting an increasing procoagulant imbalance).

They found that the factor VIII-to-protein C ratio was nonsignificantly higher in patients with simple steatosis and significantly higher in patients with nonalcoholic steatohepatitis (NASH) and in patients with metabolic or alcoholic/viral cirrhosis than in controls.

Thrombin generation in the presence/absence of thrombomodulin was significantly higher in all three groups of patients with NAFLD and in patients with alcoholic/viral cirrhosis compared with controls, and there was a progressive increase in the ETP ratio from simple steatosis or NASH to metabolic cirrhosis.

The ETP ratio correlated directly with factor VIII and the factor VIII-to-protein C ratio and inversely with antithrombin and protein C.

PICI median percentage values were nonsignificantly lower in patients with simple steatosis and significantly lower in patients with NASH and in those with metabolic or alcoholic/viral cirrhosis compared with controls. PICI% values correlated directly with antithrombin and protein C and inversely with factor VIII and the factor VIII-protein C ratio; they also correlated significantly and inversely with the ETP ratio.

The procoagulant imbalance was more marked among patients with metabolic syndrome, fibrosis, steatosis, lobar inflammation, and higher NAFLD activity scores (NAS).

"Since activated factor VIII is inhibited in vivo by activated protein C, the above results suggest that in general the procoagulant imbalance observed in NAFLD appears to result from increased factor VIII combined with decreased protein C activity," the researchers say.

"Presently, it is difficult to say what to do after finding hypercoagulability," Dr. Tripodi said. "I guess that patients would benefit from anticoagulation that should decrease the risk of cardiovascular disease and liver fibrosis. Clinical trials are needed, and our work could pave the way for such studies."

"We can start treating appropriately any disease when we understand the mechanisms behind it," he added. "We have just started to learn a bit more about NAFLD, a condition that is highly prevalent in Western countries and will become more and more prevalent if one considers the changing life style."

SOURCE: http://bit.ly/1krp8br

J Hepatol 2014.

 

Hepatitis C investigation continues

 

 

In the days since health officials announced a former hospital employee may have exposed patients to hepatitis C, more details about the situation are coming to light.

Clark County Public Health and PeaceHealth Southwest Medical Center officials announced Monday they are conducting a joint investigation into whether a former hospital employee suspected of diverting drugs at the hospital exposed any patients to the contagious, blood-borne liver disease.

So far, the investigation has not uncovered any proven instances of hepatitis C transmission to hospital patients, said Tim Strickland, a spokesman for PeaceHealth Southwest Medical Center.

But the announcement did spark questions from the community.

The day after the announcement, the hospital's patient support phone line received nearly 80 calls. Of those calls, a small percentage were from patients expressing anxiety and concern, Strickland said. Most callers asked general questions about hepatitis C and the investigation, he said.

The joint investigation is ongoing and centers around the former employee.

The employee — who hospital officials have declined to identify — began working at the hospital in the middle of 2012. In March, hospital officials had "reasonable suspicion" that the employee was diverting controlled substances, Strickland said.

"As soon as we became aware of the concern with the former employee, that person was suspended and removed from patient care," Strickland said.

The hospital did not say whether that employee had hepatitis C.

The employee left the organization in March, though hospital officials are not disclosing whether the person quit or was fired. Hospital officials aren't disclosing what position the employee held or in which department the employee worked, but Strickland said the employee did provide direct patient care.

When hospital officials learned of the possible drug diversion, they looked back at the patients for whom the employee had provided care. That's when they learned the employee was a member of the care team for a patient who, a few months after receiving care at PeaceHealth Southwest Medical Center, was diagnosed with hepatitis C, Strickland said.

The patient, who was diagnosed in late 2012, had no known risk factors for hepatitis C. At the time of the diagnosis, public health officials investigated whether any other known cases of hepatitis C in the county had a connection to PeaceHealth Southwest. They found no other cases with links to the hospital, Strickland said.

But the employee's suspected drug diversion and involvement in the patient's care prompted health officials to take another look at the case, Strickland said.

"What we're doing is acting with great caution to ensure the wellbeing of our patients," he said. "It is possible that a hepatitis C exposure happened here, but we don't know if that's the case."

As public health and hospital officials continue to comb through patient files, they will make a list of people to notify about potential exposure. Letters will be mailed to those patients no later than May 20, Strickland said.

In the meantime, hospital and public health officials are keeping law enforcement and other regulatory organizations informed of the investigation.

"Because the situation involves controlled substances and a reportable condition, we have certain notification obligations," Strickland said.

The Washington State Department of Health stipulates which type of health care providers can prescribe controlled substances — advanced registered nurse practitioners and physicians, for example — and which providers can administer the drugs ordered by authorized prescribers. Licensed practical nurses and registered nurses, for example, can administer the drugs under the direction of prescribers.

Individuals and entities that prescribe and distribute narcotics and other controlled substances are required to have a registration number with the Drug Enforcement Administration, which has regulatory oversight to ensure the drugs aren't diverted.

While the hospital has declined to identify the employee, if the circumstances of the situation were to change — if criminal charges are filed, for example — the hospital may reconsider its position, Strickland said.

 

http://www.columbian.com/news/2014/may

 

Hepatitis C vaccine: Need of the hour.

Hepatitis C virus (HCV) was first identified in 1989. HCV is a small, enveloped RNA virus. Globally, 3-4 million persons are infected with HCV each year, and are at risk of developing liver cirrhosis and/or liver cancer. The common modalities of the spread of hepatitis C infection are blood transfusions, injection drug use, unsafe therapeutic injections, and healthcare-related procedures. The standard treatment forhepatitis C has been combination antiviral therapy with interferon (IFN) and ribavirin, which are effective against all the genotypes of hepatitis viruses (pan-genotypic). A 12-month course of Peg-IFN/ribavirin treatment costs>$20 000. New HCV-specific antiviral drugs, especially in combination, have shown very high cure rates; however, the annual cost for a single subject ($82 000) make these unaffordable in most of the world. There is no hepatitis C vaccine. However, several vaccines in development, and some have shown promising preclinical results. Over the last few years, numerous HCV vaccine approaches have been assessed in mice and primates, but only a few vaccines have progressed tohuman trials. The challenge to develop HCV vaccine is to move into larger at-risk or infected populations to test efficacy.

 

http://europepmc.org/abstract/MED/24809484/reload=0;jsessionid=XUqU5c7OvE2SrtRvWxgo.24

EMA, FDA to facilitate clinical investigation of new medicines for Gaucher disease in children

Published 15 May 2014

The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have released a draft joint proposal to facilitate the clinical investigation of new medicines for the treatment of Gaucher disease in children.

The document is released for public consultation until 31 August 2014.

The aim of the proposal is not only to facilitate a rapid and smooth agreement of an EMA Paediatric Investigation Plan and FDA Pediatric Study Plan, but also to address the feasibility of developing multiple medicines for a rare disease in a reduced timeframe and in a limited number of patients.

Gaucher disease is a rare condition characterized by the accumulation of specific chemical substances (glucocerebrosides) in certain cells throughout the body. The severity of the disease is extremely variable with a wide range of symptoms. There is a high unmet medical need for children with neurological symptoms, in particular for new routes of administration that would reduce the treatment burden.

A number of medicines are under development for the treatment of this disease; however, since it is a rare condition, recruitment of children in clinical trials has been shown to be difficult.

To address this issue, the joint proposal discusses two complementary approaches:

the extrapolation of efficacy from adults to children which would include the use of modelling and simulation approaches. The EMA and FDA consider that the primary rationale for extrapolation is to avoid unnecessary studies in children for ethical reasons, to increase efficiency, to reduce the burden on children, and to ensure that resources are allocated to areas where studies are most needed;
the possibility to conduct multi-arm, multi-company clinical trials to determine the safety and efficacy of several new medicines at the same time. This approach would allow reducing the total number of children to be included in trials, compared to separate controlled trials, as the same control arm would serve more than one medicine under evaluation. A proposed design for such trials is included in the document.
"The complementary approach proposed would not only facilitate the investigation of new medicines for rare diseases in children but also optimise the number of patients recruited, thereby reducing the burden on children and families," explains Jordi Llinares Garcia, Head of the EMA's Product Development Scientific Support Department.

Sponsors who wish to make use of these innovative approaches in their development plan are advised to contact the regulatory authorities to get scientific advice. Sponsors have the possibility to approach the EMA or the FDA separately or to request parallel scientific advice from the two regulatory authorities if they wish.

The joint proposal published today is the result of an extensive consultation process with various groups of stakeholders that started in October 2011. As part of this process, the EMA and the FDA organised a joint workshop in September 2012 to gather views from patients, experts and medicines' developers to discuss the way forward for the clinical investigation of medicines for the treatment of Gaucher disease in children.

 http://www.pharmtech.com/pharmtech/News/FDA-and-EMA-Join-Forces-on-Treatment-of-Gaucher