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Specialty Pharma Industry News is provided by SPA Member Dr. Larry Boos
August 30
The U.S. Food and Drug Administration issued a Federal Register notice identifying nine bulk drug substances that the agency has considered and is proposing not to include on the 503B Bulks List of bulk drug substances (or active pharmaceutical ingredients) that outsourcing facilities may use in drug compounding. 

The nine bulk drug substances are: dipyridamole, ephedrine sulfate, famotidine, hydralazine hydrochloride, methacholine chloride, sodium bicarbonate, sodium tetradecyl sulfate, trypan blue, and vecuronium bromide.  Each of these substances is an ingredient of one or more FDA-approved drug products.

September is National Preparedness Month.  A major public health emergency like a hurricane or a lengthy power outage can limit your access to supplies and services for several days, weeks, or even months. Be prepared with safe water and food, basic supplies, and the personal items you need to protect your health in an emergency.  Visit CDC's Emergency Preparedness & Response website to learn how to prepare Personal Needs, Prescriptions, Paperwork, Power Sources, and Practical Skills.   Download PDF.
August 29
FDA scientific experts showcase cutting-edge science underpinning FDA regulatory decision-making at public Sept 11-12, 2019 FDA Science Forum entitled Transforming Health: Innovation in FDA Science.   Wed., Thurs., September 11-12.  Register to attend in person or virtually at www.fda.gov/scienceforum.
August 28
FDA warns patients and health care professionals about rare instances of serious liver injury or failure with certain hepatitis C treatments in some patients with advanced liver disease.  The U.S. Food and Drug Administration has received reports that the use of Mavyret, Zepatier, or Vosevi to treat chronic hepatitis C (HCV) in patients with moderate to severe liver impairment has resulted in rare cases of worsening liver function or liver failure. These medicines are not indicated for use in patients with moderate to severe liver impairment, but are safe and effective in patients with no or mild liver impairment.

Nature's One, Lewis Center, Ohio is recalling PediaSmart® SOY Vanilla Beverage Mix because milk is not listed under the "contains" label statement.   People who have an allergy or severe sensitivity to milk run the risk of serious or life-threatening allergic reaction if they consume this product.
August 27
The U.S. Food and Drug Administration today approved Nourianz (istradefylline) tablets as an add-on treatment to levodopa/carbidopa in adult patients with Parkinson's disease (PD) experiencing "off" episodes. An "off" episode is a time when a patient's medications are not working well, causing an increase in PD symptoms, such as tremor and difficulty walking.  According to the National Institutes of Health, PD is the second-most common neurodegenerative disorder in the U.S. after Alzheimer's disease. An estimated 50,000 Americans are diagnosed with PD each year, and about one million Americans have the condition. The neurological disorder typically occurs in people over age 60, although it can occur earlier. It happens when cells in the brain, which produce a chemical called dopamine, become impaired or die. Dopamine helps transmit signals between the areas of the brain that produce smooth, purposeful movements - such as eating, writing, and shaving. Early symptoms of the disease are subtle and typically worsen gradually; however, the disease progresses more quickly in some people than in others. 

The FDA is reminding patients and health care professionals not to use drug products intended to be sterile made by Pacifico National Inc., an outsourcing facility doing business as AmEx Pharmacy, in Melbourne, Florida. The drugs-which include compounded ophthalmic products among other drug products - pose unnecessary risks due to significant quality and sterility concerns.  An FDA inspection of AmEx Pharmacy's facility found conditions that could cause the company's drugs to become contaminated or otherwise pose risks to patients. 

FDA requests recall of sterile compounded drug products by AmEx Pharmacy and reminds patients and health professionals to stop using due to potential risks.  An FDA inspection of AmEx Pharmacy's facility found conditions that could cause the company's drugs to become contaminated or otherwise pose risks to patients. Administration of a non-sterile drug intended to be sterile presents the risk of infection. Intraocular medications which are not sterile can lead to intraocular infections such as endophthalmitis which, depending on the infecting microorganism, can lead to a loss of partial or total permanent visual function (i.e., no light perception).
August 26
The U.S. Food and Drug Administration (FDA) is announcing the availability of a draft guidance for industry titled, " Male Breast Cancer: Developing Drugs for Treatment."
This draft guidance provides recommendations to sponsors regarding the development and labeling of cancer drugs, including biological products, for the treatment of male patients with breast cancer. Males have historically been excluded from clinical trials of breast cancer drugs because breast cancer in males is rare. As a result, there are limited FDA-approved treatment options specifically approved for males with breast cancer.
August 25
Treating Nonmetastatic Breast Cancer in 2019.  Breast cancer outcomes continue to improve. Treatments for the disease are very effective and continually evolving. We spoke with Patricia A. Ganz, MD, from UCLA about what is new in breast cancer treatment. Start Learning
August 23
US FDA reports fake inspection documents of Chinese OTC manufacturer.  FDA published a warning letter to Ningbo Huize Commodity Co., a Chinese manufacturer of OTC drugs. The case concerns serious violations of good manufacturing practice and, in this context, the serious manipulation of data. During an FDA inspection in March 2019, the company handed over various quality control documents, which the managing director now admitted had been forged specifically for inspection. These include, for example, reports on cleaning validation and also batch records and other control documents for the company's various products. Records of the regularly required "Quality Reviews" were also falsified. These cGMP violations result in all drugs manufactured by Ningbo Huize being considered counterfeit under the Federal Food, Drug, and Cosmetic Act (FD&C Act).
August 22
Warning Letters
Dr. Enrique Feoli, Technical Director, Polimeros y Servicios S.A. Tibas, San Jose 11305, Costa Rica
During our inspection, our investigator observed specific violations including, but not limited to, the following.
1. Your firm failed to have, for each batch of drug product, appropriate laboratory determination of satisfactory conformance to final specifications for the drug product, including the identity and strength of each active ingredient, prior to release.
2. Your firm failed to conduct at least one test to verify the identity of each component of a drug product. Your firm also failed to validate and establish the reliability of your component supplier's test analyses at appropriate intervals.
3. Your firm failed to assure that the drug product bore an expiration date that was supported by appropriate stability testing
4. Your firm failed to establish and follow adequate written procedures for cleaning and maintenance of equipment.


Mr. Huaijun Yin, Co-Owner, Yino, Inc. Yubei Qu Chongqing Shi 401120, China  Because your methods, facilities, or controls for manufacturing, processing, packing, or holding do not conform to CGMP, your API are adulterated within the meaning of section 501(a)(2)(B) of the Federal Food, Drug, and Cosmetic Act. We have not received a response to our Form FDA 483 from your firm for corrective actions to the observations identified during the inspection.
1. Failure to transfer all quality or regulatory information received from the API manufacturer to your customers.
2. Failure to establish, document, and implement an effective system to manage quality. For example, the certificate of analysis (COA) created and issued by your firm lacked a signature and date from an authorized person in the Quality Unit.


 Mr. Wee Ee Lim, CEO, Haw Par Healthcare Limited, Singapore 149598, Singapore  During our inspection, our investigator observed specific violations including, but not limited to, the following.
1. Your firm failed to thoroughly investigate any unexplained discrepancy or failure of a batch or any of its components to meet any of its specifications, whether or not the batch has already been distributed.
2. Your firm failed to establish laboratory controls that include scientifically sound and appropriate specifications, standards, sampling plans, and test procedures designed to assure that components, drug product containers, closures, in-process materials, labeling, and drug products conform to appropriate standards of identity, strength, quality, and purity.
3. Your firm failed to ensure that laboratory records included complete data derived from all tests necessary to ensure compliance with established specifications and standards.
4. Your firm failed to use equipment in the manufacture, processing, packing, or holding of drug products that is of appropriate design, adequate size, and suitably located to facilitate operations for its intended use and for its cleaning and maintenance.

August 19
The U.S. Food and Drug Administration today approved Xenleta (lefamulin) to treat adults with community-acquired bacterial pneumonia. Community-acquired pneumonia occurs when someone develops pneumonia in the community (not in a hospital). Pneumonia is a type of lung infection that can range in severity from mild to severe illness and can affect people of all ages. The safety and efficacy of Xenleta, taken either orally or intravenously, was evaluated in two clinical trials with a total of 1,289 patients with CABP. In these trials, treatment with Xenleta was compared to another antibiotic, moxifloxacin with or without linezolid. The trials showed that patients treated with Xenleta had similar rates of clinical success as those treated with moxifloxacin with or without linezolid.
August 16
Recently Posted Guidance Documents

August 15
FY 2020 PDUFA Fees and Program Invoice Update The fiscal year (FY) 2020 PDUFA Program Fee invoices were emailed on August 15, 2019. Please note that the invoices are only sent out to firms which have PDUFA user fee eligible products. If you do not receive your invoice by August 15, 2019, and believe you should receive a program fee invoice, please contact PDUFA User Fee staff by email at [email protected].
August 2, 2019. The fee rates for FY 2020 are shown below:

  User Fee Type
FY 2020
FY 2019
  Application Fee - Clinical Data Required
  $2,942,965
  $2,588,478
  Application Fee - No Clinical Data Required
$1,471,483
$1,294,239
  Program Fee
$325,424
$309,915









Pfizer Inc. is voluntarily recalling RELPAX® (eletriptan hydrobromide) 40 mg tablets, lots AR5407 and CD4565, to the Patient level. Pfizer Inc. initiated this recall because these product lots may not meet Pfizer's in-house microbiological specification for the potential presence of Genus Pseudomonas and Burkholderia.  Individuals who consume oral products contaminated with microorganisms are at risk of bacterial dissemination from the gut to the bloodstream potentially resulting in serious, life- threatening infections. In addition, there is risk of temporary gastrointestinal distress without serious infection.
August 14
FDA approves new drug for treatment-resistant forms of tuberculosis that affects the lungs  The U.S. Food and Drug Administration today approved Pretomanid Tablets in combination with bedaquiline and linezolid for the treatment of a specific type of highly treatment-resistant tuberculosis (TB) of the lungs.   The safety and effectiveness of Pretomanid, taken orally in combination with bedaquiline and linezolid, was primarily demonstrated in a study of 109 patients with extensively drug-resistant, treatment-intolerant or non-responsive multidrug-resistant pulmonary TB (of the lungs). Of the 107 patients who were evaluated six months after the end of therapy, 95 (89%) were successes, which significantly exceeded the historical success rates for treatment of extensively drug resistant TB.

Publication of Level 2 guidance revision: Questions and Answers on Current Good Manufacturing Practices - Laboratory Controls      The FDA has added three new questions and answers, numbers 15 - 17, to the Laboratory Controls section of the Questions and Answers on Current Good Manufacturing Practices (CGMP) guidance. The new questions and answers discuss instrument calibration standards for chromatographic systems, system suitability, and trial injections.    Because drug manufacturers rely, in part, on laboratory testing to verify the quality of drugs before introduction into the market place (or further manufacture), it is essential that analytical procedures are correct and strictly followed. The accuracy of test results rely on appropriate use of standards and prepared samples in executing the test method. The Q&As are intended to make clear how standards and samples should and should not be used in chromatographic testing.
August 13
Aspirin use may be widespread despite new guidelines.  A study suggests that millions of U.S. adults may take a daily aspirin for cardiovascular disease prevention without any clear benefit.  Of the people who didn't have cardiovascular disease, about 23% were taking a daily aspirin for prevention. Nearly 23% of these took a daily aspirin without a health care provider recommendation. Nearly half of the survey participants who were 70 years or older and did not have heart disease reported daily aspirin use. A quarter of people who had a history of stomach ulcers, but not cardiovascular disease, also used aspirin.  Studies have shown that aspirin can help some people prevent a second heart attack or stroke. However, taking aspirin also comes with a higher risk of bleeding.  The findings suggest that people speak with their health care providers before taking aspirin daily.

Miracle Mineral Solution (MMS): FDA warns consumers about the dangerous and potentially life threating side effects.  The FDA is warning consumers not to purchase or drink the product sold online as a medical treatment due to a recent rise in reported health issues.  The solution, when mixed, develops into a dangerous bleach which has caused serious and potentially life-threatening side effects.  Since 2010, the FDA has warned consumers about the dangers of Miracle or Master Mineral Solution, Miracle Mineral Supplement, MMS, Chlorine Dioxide Protocol, Water Purification Solution and other similar products. Miracle Mineral Solution has not been approved by the FDA for any use, but these products continue to be promoted on social media as a remedy for treating autism, cancer, HIV/AIDS, hepatitis and flu, among other conditions.

FDA today announced the availability of a final guidance for industry entitled "Child-Resistant Packaging Statements in Drug Product Labeling."  This guidance will assist applicants, manufacturers, packagers, and distributors who choose to include child-resistant packaging (CRP) statements in their drug product labeling.
August 9
Ridge Properties LLC DBA Pain Relief Naturally is voluntarily recalling 4 lots of 4% lidocaine topical cream & liquid gel products sold at the consumer level. FDA analysis has found these products to have microbiological contamination and the potency is higher than the labeled amount for lidocaine.

FDA is announcing user fee rates for fiscal year 2020 for prescription drugs, generic drugs, biosimilars, and outsourcing facilities.   View the web page.

The link between pot and mass shootings may be closer than we think.   Read here.  
August 8
NIH News in Health:  What to Do About Varicose Veins.  Varicose veins aren't just a cosmetic issue. If they aren't treated, they can also cause health problems. Read more here:  the August 2019 edition of NIH News in Health. 
August 1
New CDC Online Resource: What Is Dementia?   Get the facts on what is dementia, the various types of dementia, and action steps you can take if you or a loved one shows signs of dementia.