Chest Pain or Angina Pectoris
Angina pectoris is the medical name for chest pain or discomfort due to a disease in the heart’s arteries.
Who gets it?
Atherosclerosis causes a narrowing of the heart’s blood vessels. If more than 50% of the artery gets blocked, angina pectoris may start.
What causes it?
Our heart is a muscle. Muscles in our body, like any other organ, need oxygen in order to function. They get that oxygen when our blood, which is rich in oxygen, reaches them.
When the arteries which supply the heart with blood (called coronary arteries) are narrowed, like in atherosclerosis, the heart doesn’t get enough blood. In medicine, an insufficient blood supply is called ischemia. This is why you may hear the term “ischemic heart disease”, for the condition caused by atherosclerosis.
When the heart doesn’t get enough blood it “shouts” at you by producing pain, as you’ll see in the “how does it feel” section.
Angina occurs when our arteries are narrowed enough by a plaque that not enough blood reaches the heart.
How does it feel?
Angina is manifested as chest pain. It’s usually felt under the breast bone (around the center of the chest) like a deep pain. It usually lasts less than 30 minutes, and can radiate to the neck, the jaw or the left arm. It can be accompanied by breathlessness, cold sweat and nausea or vomiting.
There are 2 types of angina:
- 1. Stable angina – These occur when you exert yourself (such as when walking or running) or when you’re under mental or emotional stress. Normally the pain subsides when you rest or when you take a drug called nitroglycerin.
- 2. Unstable angina – This occurs when the narrowing of the coronary arteries is more severe. In this stage, the pain occurs even when you rest, meaning you don’t even have to have your heart pumping stronger than usual for it to feel the lack of oxygen – it will feel it even when it pumps regularly. This is a dangerous situation which signals a possible upcoming heart attack.The pain here can last longer and be more painful than in stable angina.
How is it discovered?
There are a few things a doctor can do to diagnose angina –
- 3. The typical story helps to point in the right direction. Also doing a physical exam will help.
- 4. An ECG (electrocardiogram) is done – This is a graph showing the electrical activity of the heart. To have this graph drawn, electrodes are attached to your chest, which detect the heart’s activity. This isn’t painful at all. When the heart lacks oxygen, a pattern can be seen on the ECG which the doctor will recognize.
Example of an ECG graph.
- 5. Stress testing – Like mentioned above, angina doesn’t occur all the time, and sometimes a person with angina will come to the doctor when they don’t have pain at the moment. This is why stress testing is done. In it you run on a treadmill (like in a gym), causing your heart to work harder. This will trigger the pain of angina and the ECG changes, if your arteries are narrow. (There are people who can’t exercise for whatever reason. For these people a drug is given which makes their heart work harder, causing the same effect).
- 6. Coronary angiography – When there is a high suspicion that you are at risk for a heart attack, or when the other tests are positive, a coronary angiography (also called catheterization) may be done. In this exam, a small tube-like device called a catheter is inserted through large arteries in the body, and reaches the coronary arteries. There it injects a material which can be seen in real time under X-ray. This shows the coronary arteries, and will show if they are narrowed.
The heart’s arteries (coronary arteries) as seen in a coronary angiography. Photo by Bleiglass.
How is it treated?
There are a few things that can be done to treat this condition:
- 7. Changing risk factors which are reversible – Such as quitting smoking, lowering blood pressure, changing the diet. (For a complete list of the risk factors, see part 1).
- 8. Drugs – There are several drugs given in this condition –
a. Antiplatelet drugs – As you will see in part 3, angina can be a step before a heart attack. Since platelets are involved in the development of heart attacks (again, you’ll see why in part 3), inhibiting their action can help prevent a heart attack from happening. An example of such a drug is aspirin.
b. Beta-blockers – These are drugs that make the heart work less strenuously, lowering the chance that it will reach a point where it lacks oxygen.
c. Nitroglycerin – This drug causes widening of blood vessels, causing more blood to reach the heart. It’s usually taken when needed – when there’s pain.
d. Statins – These are drugs that take down the “bad cholesterol” (LDL cholesterol) in the blood.
There are also other drugs which can be used, but the ones above are the most common.
If the narrowing is severe or if the above treatment doesn’t help, procedures for opening the narrowing in the heart’s arteries are performed.
What happens after treatment?
When not treated, heart attacks are likely to develop in people with angina, putting you at risk for death. When treated, however, this condition can be followed, with the chances of having a heart attack being greatly reduced.
Every year, more than 1 million Americans have a heart attack – a sudden interruption in the heart’s blood supply. This happens when there is a blockage in the coronary arteries, the vessels that carry blood to the heart muscle. When blood flow is blocked, heart muscle can be damaged very quickly and die. Prompt emergency treatments have reduced the number of deaths from heart attacks in recent years.
This section deals with the real thing – the heart attack that can be caused by the plaques in the heart’s coronary arteries.
Danger level: High
What is it?
A heart attack (or myocardial infarction by its medical name) occurs when the blood supply to part of the heart is interrupted, causing the heart cells in that part to die.
Who gets it?
People in danger of having a heart attack are the same ones who have the risk factors for developing atherosclerosis
What causes it?
The plaque that builds up inside the artery can eventually burst, tear or rupture. When the plaque ruptures, the body recognizes this as damage to the artery. Remember what happens when you have a wound on your skin? Within a short time it clots, sealing down the wound so that blood won’t spill from it. The same thing happens here: The “wound” in the artery clots.
How does this clotting happen? Platelets are the cells in our blood responsible for clotting. They aggregate together, helping the clot form. The clot is called a thrombus.
You might think to yourself – “the body has done its job right. The wound in the artery is healed.”. But the clotting process here is actually what brings about the heart attack. The clot in the artery can lead to an occlusion of the blood flow inside the artery, causing blood to not pass in the artery anymore. This leads to blood not reaching where it should in the heart.
When blood doesn’t reach a part of the heart, that part doesn’t get oxygen. When this happens, the cells in that part start dying. When they finally die, they can’t grow back, and that part of the heart is lost and will not function anymore.
The time it takes for the cells to die is a few hours. If the condition is treated within that time frame, the heart can be saved.
This video shows the process visually –
How does it feel?
A heart attack feels like angina, but lasts more than 30 minutes and is not relieved by nitroglycerine or by rest. Here’s a reminder of the symptoms:
- Pain, pressure, discomfort or heaviness in the chest. It can radiate to the left arm, the neck or the jaw.
- Sweating, nausea, vomiting or dizziness may accompany the pain.
- Rapid or irregular heartbeats may be felt as well.
How is it discovered?
There are a few things that can be done when you reach the ER to see if you have a heart attack:
- ECG: You can read an explanation about that in part 2.
- Blood tests: When the cells in the heart start dying, certain materials (called enzymes) start leaking out from them and into the blood. These enzymes are called troponins. When their level in the blood is high, this hints to a heart attack.
- Coronary angiography – (see an explanation in part 2) can be used to visualize the arteries in the heart to see if they are blocked.
How is it treated?
Perfusion is a word that means blood coming into an organ. In a heart attack, blood is not reaching the heart muscle properly. In order to bring blood back into the heart, the artery needs to be re-opened. This process is called re-perfusion.
There are 3 ways to get the artery opened:
- Percutaneous coronary intervention (PCI) – This is a long and complicated name, but the procedure is simple to understand. Angiography show your heart’s arteries?
- It can also be used to treat the arteries. The catheter which is inserted into the arteries is used to do one of two things. It can inflate a balloon, which will open the block in the artery. Then it can insert something called a stent – which is a tube inserted into the artery to keep it open.
This video shows how it works:
- Thrombolysis – In this procedure a material is injected into the blood vessels, which causes the clot to “melt”. This opens the heart’s arteries back. While it sounds easier than the first method, it’s not as efficient, and not everyone is suitable for this procedure.
- Coronary artery bypass surgery (CABG) – Sometimes neither of the first two treatments fit. In this setting, a surgery has to be performed. In it, the chest is opened and the heart is exposed. The surgeon then takes an artery or vein from somewhere else in the body and implants it to the heart’s arteries. In this way, the occlusion is bypassed by a new, open, blood vessel.
Besides those, some drugs are given to everyone having a heart attack. They include aspirin (which stops the platelets from aggregating and so slows down the process), pain killers, oxygen and others.
What happens after treatment?
Without treatment, death rates are high for people who undergo a heart attack. But even with treatment, death can occur, especially before reaching the hospital. This is mainly due to complications that can occur as part of the heart attack, such as problems with the heart rhythm, failure of the heart’s muscle to pump properly, problems with the heart’s valves and others.
The bottom line – How do I avoid it?
The best way to avoid a heart attack is to prevent the process of atherosclerosis from happening.
If you do have atherosclerosis or if you had a heart attack before, you can prevent it from happening by visiting your doctor. They will prescribe some drugs for you (including statins, beta blockers, aspirin, and others. Taking these drugs regularly will lower your chances of getting a heart attack.
Patent Office Issues Notice of Allowance For eSource Software
VERY BIG NEWS FOR THE PHARMACEUTICAL INDUSTRY – GOODBYE PAPER RECORDS
Target Health has received a Notice of Allowance from the US Patent Office for Target e*CTR® (Target e*Clinical Trial Record). Target e*CTR is the electronic clinical trial patient record, access to which is controlled by the clinical investigator. Target e*CTR is created at the time data are initially entered into any electronic data base, but before the data are entered into the sponsor’s or EDC database. Therefore, Target e*CTR can be considered the original patient record (source document) associated with the patient record when direct data entry (DDE) occurs at the patient visit. Target e*CTR can be integrated seamlessly with any EDC and EHR system and does NOT affect any end-user or workflow of any EDC system, thus does not require any training. A ROI analysis indicates a minimum savings of $6,000 per clinical site if monitoring visits drop from 5 to 3 in a 1-year study, and $10,000/site if visits drop from 6 to 3.
Target Health just finished its first study using Target e*CTR and is starting its next study this month. An NDA planned Q4 2012. The following are the results of the first study:
- Screening errors picked up early
- Real time monitoring at the time of data entry
- No need to do additional subject recruitment
- EDC edit checks modified early in the game
- Compliance issues identified in real time
- Immediate availability of safety issues
- Site saved 70 hours of data entry time
- Nothing to do after patient left the clinic
For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 104). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel or Ms. Joyce Hays. The Target Health software tools are designed to partner with both CROs and Sponsors. Please visit the Target Health Website at www.targethealth.com
Protein Switches Could Turn Cancer Cells Into Tiny Chemotherapy Factories
Brain Cancer Cell
Breast Cancer Cell
Johns Hopkins researchers have devised a protein “switch” that instructs cancer cells to produce their own anti-cancer medication. In lab tests, the researchers showed that these switches, working from inside the cells, can activate a powerful cell-killing drug when the device detects a marker linked to cancer. The goal, the scientists said, is to deploy a new type of weapon that causes cancer cells to self-destruct while sparing healthy 1) ___.
This new cancer-fighting strategy and promising early lab test results were reported this week in the online early edition of Proceedings of the National Academy of Sciences (DOI: 10.1073/pnas.1102803108). Although the switches have not yet been tested on 2) ___ patients, and much more testing must be done, the researchers say they have taken a positive first step toward adding a novel weapon to the difficult task of treating cancer.
One key problem in fighting cancer is that broadly applied chemotherapy usually also harms healthy 3) ___. In the protein switch strategy, however, a doctor would instead administer a “prodrug,” meaning an inactive form of a cancer-fighting drug. Only when a cancer marker is present would the cellular switch turn this harmless prodrug into a potent form of chemotherapy. “The switch in effect turns the cancer cell into a factory for producing the anti-cancer 4) ___ inside the cancer cell,” said Marc Ostermeier, a Johns Hopkins chemical and biomolecular engineering professor in the Whiting School of Engineering, who supervised development of the switch. “The healthy cells will also receive the 5) ___,” he added, “and ideally it will remain in its non-toxic form. Our hope is that this strategy will kill more cancer cells while decreasing the unfortunate side effects on healthy cells.”
To demonstrate that these switches can work, the research team successfully tested them on human colon cancer and breast 6) ___ cells in Ostermeier’s lab and in the laboratory of James R. Eshleman, a professor of pathology and oncology in the Johns Hopkins School of Medicine. “This is a radically different tool to attack cancers,” said Eshleman, a co-author of the PNAS journal article, “but many experiments need to be done before we will be able to use it in patients.” The next step is animal testing, expected to begin within a year, Ostermeier said.
Ostermeier’s team made the cancer-fighting switch by fusing together two different proteins. One protein detects a marker that cancer cells produce. The other 7) ___, from yeast, can turn an inactive prodrug into a cancer-cell killer. “When the first part of the switch detects cancer, it tells its partner to activate the chemotherapy drug, destroying the cell,” Ostermeier said. In order for this switch to work, it must first get inside the cancer cells. Ostermeier said this can be done through a technique in which the switch gene is delivered inside the cell. The switch 8) ___ serves as the blueprint from which the cell’s own machinery constructs the protein switch. Another approach, he said, would be to develop methods to deliver the switch protein itself to cells. Once the switches are in place, the patient would receive the inactive chemotherapy drug, which would turn into a cancer attacker inside the cells where the switch has been flipped 9) ___.
Although many researchers are developing methods to deliver anti-cancer drugs specifically to cancer cells, Ostermeier said the protein switch tactic skirts difficulties encountered in those methods. “The protein 10) ___ concept changes the game by providing a mechanism to target production of the anti-cancer drugs inside cancer cells instead of targeting delivery of the anti-cancer drug to cancer cells,” he said.
ANSWERS: 1) tissue; 2) human; 3) cells; 4) drug; 5) prodrug; 6) cancer; 7) protein; 8) gene; 9) on; 10) switch
Urine and Medicine
A Doctor Examining Urine — Date painted: First half of 17th Century
Many physicians in history have resorted to the inspection and examination of the urine of their patients. Hermogenes, a Greek physician who probably lived in the third or second century BCE,wrote about the color and other attributes of urine as indicators of certain diseases. Abdul Malik Ibn Habib of Andalusia(southern Iberia or Spain) d.862 CE, mentions numerous reports of urine examination throughout the Umayyad empire. Diabetes mellitus got its name because the urine is plentiful and sweet.
A urinalysis is a medical examination of the urine and part of routine examinations. A culture of the urine is performed when a urinary tract infection is suspected. A microscopic examination of the urine may be helpful to identify organic or inorganic substrates and help in the diagnosis. The color and volume of urine can be reliable indicators of hydration level. Clear and copious urine is generally a sign of adequate hydration, dark urine is a sign of dehydration. The exception occurs when alcohol, caffeine, or other diuretics are consumed, in which case urine can be clear and copious and the person still be dehydrated.
In Roman times, there was a tradition among the Gauls to use urine to brush and whiten teeth. This practice lasted into the Renaissance. Ancient Romans used human urine to cleanse grease stains from their clothing, before acquiring soaps from the Germans during the first century CE. Urine that has been fermented for the purposes of cleaning is referred to as lant. The emperor Nero instituted a tax (Latin: vectigal urinae) on the urine industry. This tax was continued by Nero’s successor, Vespasian, to whom is attributed the Latin saying Pecunia non olet (money doesn’t smell) – this is said to have been Vespasian’s reply to a complaint from his son about the disgusting nature of the tax. Vespasian’s name is still attached to public urinals in France (vespasiennes), Italy (vespasiani), and Romania (vespasiene).
From the first millennium BCE to about 500 CE, foundational works of religious Sanskrit text contain stanzas on the benefits of “pure water, or one’s own urine”. In this text, urine therapy is referred to as Shivambu Kalpa. This ancient Indian text suggests, among other uses and prescriptions, massaging one’s skin with fresh, concentrated urine. Alchemists spent much time trying to extract gold from urine, and this effort led to discoveries such as white phosphorus, which was discovered by the German alchemist Hennig Brand in 1669 when he was distilling fermented urine. In 1773 the French chemist Hilaire Rouelle discovered the organic compound urea by boiling urine dry.
The word “urine” was first used in the 14th century. Before that, the concept was described by the now vulgar word “piss”. Onomatopoetic in origins, “piss” was the primary means of describing urination, as “urinate” was at first used mostly in medical contexts. Likely, “piss” became vulgar through its use by lower class characters such as the Reeve and the Wife of Bath in Geoffrey Chaucer’s 14th century work The Canterbury Tales.
The last great Byzantine physician was John Actuarius, who lived in the early 14th Century in Constantinople. His works on urine laid much of the foundation for later study in that field. However, from the latter 12th Century to the end in 1453, there is very little outpouring in medical knowledge, largely due to the turmoil the Empire was facing on both fronts, following its resurrection after the Roman Empire and the dwindling population of Constantinople due to plague and war.
One standard authority for drugs in Europe until the end of the eighteenth century was Lamery’s Dictionnaire Universelle des Drogues. One of the late editions, published in 1759, contains a list of remedies under “Homo.” It explains: “All parts of man, his excrescences and excrements, contain oil and the sals volatile, combined with phlegm and earth…” The book recommends the drinking of two or three glasses of urine each morning to cure gout, to relieve obstructions of the bowels, and to dispel hysterical vapors.
In the early 1800s, a book titled One Thousand Notable Things describes the use of urine to cure scurvy, relieve skin itching, cleanse wounds, and many other treatments. An 18th century French dentist praised urine as a valuable mouthwash. In England during the 1860-70s, the drinking of one’s own urine was a common cure for jaundice. In more modern times, the Alaskan Eskimos have used urine as an antiseptic to treat wounds.
In China, the urine of young boys was regarded as a curative. In southern China, babies’ faces were washed with urine to protect the skin. In France, a cure for strep throat was to soak stockings in urine and wrap them around their necks. Aristocratic French women in the 17th century reportedly bathed in urine to beautify their skin. In Sierra Madre, Mexico, farmers prepared poultices for broken bones by having a child urinate into a bowl of powdered charred corn. The mixture was made into a paste and applied to the skin.
Intranasal Insulin Therapy for Alzheimer Disease and Amnestic Mild Cognitive Impairment
According to an article published online in the Archives of Neurology 12 September 2011, a randomized, double-blind, placebo-controlled clinical trial was performed at a clinical research unit of a Veterans Affairs medical center in order to examine the effects of intranasal insulin administration on cognition, function, cerebral glucose metabolism, and cerebrospinal fluid biomarkers in adults with amnestic mild cognitive impairment or Alzheimer disease (AD).
The study sample consisted of 104 adults with amnestic mild cognitive impairment (n = 64) or mild to moderate AD (n = 40) who received placebo (n = 30), 20 IU of insulin (n = 36), or 40 IU of insulin (n = 38) for 4 months, administered with a nasal drug delivery device (Kurve Technology, Bothell, Washington). The primary outcome measure was delayed story recall score and the Dementia Severity Rating Scale score, and the secondary measures included the Alzheimer Disease’s Assessment Scale–cognitive subscale (ADAS-cog) score and the Alzheimer’s Disease Cooperative Study–activities of daily living (ADCS-ADL) scale. A subset of participants underwent lumbar puncture (n = 23) and positron emission tomography with fludeoxyglucose F 18 (n = 40) before and after treatment.
Results showed that treatment with 20 IU of insulin improved delayed memory (P <0.05), and both doses of insulin (20 and 40 IU) preserved caregiver-rated functional ability (P <0.01). Both insulin doses also preserved general cognition as assessed by the ADAS-cog score for younger participants and functional abilities as assessed by the ADCS-ADL scale for adults with AD (P <.005).
Cerebrospinal fluid biomarkers did not change for insulin-treated participants as a group, but, in exploratory analyses, changes in memory and function were associated with changes in the AB42 level and in the tau protein-to-AB42 ratio in cerebrospinal fluid. Placebo-assigned participants showed decreased fludeoxyglucose F 18 uptake in the parietotemporal, frontal, precuneus, and cuneus regions and insulin-minimized progression. No treatment-related severe adverse events occurred.
According to the authors, these results support longer trials of intranasal insulin therapy for patients with amnestic mild cognitive impairment and patients with AD.
Hospitalizations Increase for Alcohol and Drug Overdoses
If we really want to reduce health care costs, we must address many of our self-inflicted diseases.
According to an article published in the Journal of Studies on Alcohol and Drugs (2011;72:774–786), hospitalizations for alcohol and drug overdoses – alone or in combination – increased dramatically among 18- to 24-year-olds between 1999 and 2008. Over the 10-year study period, hospitalizations among 18-24-year-olds increased by 25% for alcohol overdoses; 56% for drug overdoses; and 76% for combined alcohol and drug overdoses. The study examined hospitalization data from the Nationwide Inpatient Sample, a project of the U.S. Agency for Healthcare Research and Quality designed to approximate a 20% sample of U.S. community hospitals.
According to the authors, in 2008, 1 out of 3 hospitalizations for overdoses in young adults involved excessive consumption of alcohol and that alcohol overdoses alone caused 29,000 hospitalizations, combined alcohol and other drug overdoses caused 29,000, and drug overdoses alone caused another 114,000. The cost of these hospitalizations now exceeds $1.2 billion per year just for 18-24-year-olds.
According to the authors, this is a growing problem for those outside of the 18-24 age range, as well as among the entire population 18 and older, 1.6 million people were hospitalized for overdoses in 2008, at a cost of $15.5 billion, and half of these hospitalizations involved alcohol overdoses.
The current study also showed an increase of 122% in the rate of poisonings from prescription opioid pain medications and related narcotics among 18-24 year olds. An alcohol overdose was present in 1 of 5 poisonings on these medications.
The authors noted that the steep rise in combined alcohol and drug overdoses highlights the significant risk and growing threat to public health of combining alcohol with other substances, including prescription medications. They call for stronger efforts to educate medical practitioners and the general public about the dangers of excessive alcohol consumption alone or in combination with other drugs.
Gestational Age at Birth and Mortality in Young Adulthood
Preterm birth is the leading cause of infant mortality in developed countries, but the association between gestational age at birth and mortality in adulthood remains unknown. As a result, a study published in the Journal of the American Medical Association (2011;306:1233-1240) was performed to examine the association between gestational age at birth and mortality in young adulthood.
For the study, data were derived from the national cohort study of 674,820 individuals born as singletons in Sweden in 1973 through 1979 who survived to age 1 year, including 27,979 born preterm (gestational age <37 weeks), followed up to 2008 (ages 29-36 years). The main outcome measures were all-cause and cause-specific mortality.
A total of 7,095 deaths occurred in 20.8 million person-years of follow-up. Among individuals still alive at the beginning of each age range, a strong inverse association was found between gestational age at birth and mortality in early childhood (ages 1-5 years: adjusted hazard ratio [aHR] for each additional week of gestation, 0.92; P <0.001), which disappeared in late childhood (ages 6-12 years: aHR, 0.99) and adolescence (ages 13-17 years: aHR, 0.99) and then reappeared in young adulthood (ages 18-36 years: aHR, 0.96; P <0.001). In young adulthood, mortality rates (per 1000 person-years) by gestational age at birth were 0.94 for 22 to 27 weeks, 0.86 for 28 to 33 weeks, 0.65 for 34 to 36 weeks, 0.46 for 37 to 42 weeks (full-term), and 0.54 for 43 or more weeks. Preterm birth was associated with increased mortality in young adulthood even among individuals born late preterm (34-36 weeks, aHR, 1.31; P <0.001), relative to those born full-term. In young adulthood, gestational age at birth had the strongest inverse association with mortality from congenital anomalies and respiratory, endocrine, and cardiovascular disorders and was not associated with mortality from neurological disorders, cancer, or injury.
According to the authors, after excluding earlier deaths, low gestational age at birth was independently associated with increased mortality in early childhood and young adulthood.
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FDA Approves Soliris for Atypical Hemolytic Uremic Syndrome (aHUS)
The FDA has approved Soliris (eculizumab; Alexion Pharmaceuticals) for the treatment of patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke. aHUS accounts for 5-10% of all cases of aHUS. The disease disproportionately affects children.
Soliris is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA first approved Soliris in March 2007 to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare type of blood disorder that can lead to disability and premature death. Soliris is classified as an orphan drug. Orphan drugs are those that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
There are no other FDA-approved treatments for aHUS, and the safety and effectiveness of current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well controlled trials. According to FDA, this is the first approval of a drug for treating this life-threatening disease, and the first approval for use of Soliris in children, and that this approval underscores how an increased understanding of the biology of a disease and of how a drug interacts with that process can expedite drug development.”
Soliris’ safety and effectiveness were established in two single-arm trials in 37 adults and adolescent patients with aHUS and one retrospective study in 19 pediatric patients and 11 adult patients with aHUS. Patients treated with Soliris in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients that did not respond to plasma therapy. Patients treated with Soliris also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.
The most common side effects seen in patients treated with Soliris for aHUS included high blood pressure (hypertension), diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and a decrease in white blood cells (leukopenia).
This new indication for Soliris is being approved with an extension of the existing Risk Evaluation and Mitigation Strategy (REMS), to inform health care professionals and patients about the known risk of life-threatening meningococcal infections. Soliris will continue to be available only through a restricted program, and prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.
Soliris was reviewed under the FDA’s priority review program, which provides for an expedited six-month review of drugs that may offer major advances in treatment or that provide a treatment when no adequate therapy exists. The therapy also is being approved under the FDA’s accelerated approval program, designed to provide patients with earlier access to promising new drugs followed by further studies to confirm the drug’s clinical benefit. The accelerated approval program allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect on a clinical endpoint other than survival or irreversible morbidity.
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