Category Archives: Food and Drug Administration (FDA)

U.S. Food and Drug Administration (FDA) Grants Breakthrough Therapy Designation to Finch Therapeutics Investigational Drug CP101 for Treatment of Recurrent Clostridium difficile Infection (rCDI)

Finch Therapeutics Group, Inc., a clinical-stage microbiome therapeutics company, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to investigational drug CP101 for the treatment of patients with recurrent Clostridium difficile (C. difficile) infection. Breakthrough Therapy Designation is intended to expedite the development and review of investigational therapeutics for serious or life-threatening conditions where preliminary clinical evidence indicates that the product may demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints.

Finch’s lead therapeutic candidate CP101 is designed to prevent recurrent C. difficile, a bacterial infection affecting over 500,000 patients each year and leading to an estimated 29,000 annual deaths. Recurrent C. difficile has been named an urgent public health threat by the Centers for Disease Control (CDC) and, with a high percentage of patients failing standard-of-care antibiotic treatment, presents a clear and urgent unmet medical need.

“We are thrilled that CP101 has been designated as a Breakthrough Therapy for recurrent C. difficile,” said Mark Smith, CEO of Finch. “CP101 is designed to break the cycles of infection by restoring the balance of the gut microbiome, an approach supported by numerous clinical studies and Finch’s extensive experience providing microbial treatments to patients suffering from C. difficile. This designation will accelerate our efforts to provide an effective therapy for patients living with this devastating infection, and we look forward to working closely with the FDA to advance that mission.”

Finch is actively enrolling patients with recurrent C. difficile in PRISM3, a randomized, placebo-controlled Phase II clinical study to assess the safety and efficacy of CP101. The study drug is an oral capsule that is administered in a single dose. For more information about this trial, please visit www.prism3trial.com.

CP101 is not approved in any country.The FDA’s Breakthrough Therapy Designation does not constitute or guarantee a future approval and does not alter the standards for approval.

About Finch Therapeutics Group, Inc.Finch Therapeutics Group, Inc. (Finch) is developing novel microbial therapies to serve patients with serious unmet medical needs. Built on 30 years of translational research at OpenBiome, MIT, University of Minnesota and the Center for Digestive Diseases, Finch uses  Human-First Discovery  to develop therapies from microbes that have demonstrated clinically significant impacts on patient outcomes. Finch is unique in having both a donor-derived  Full-Spectrum Microbiota  ( FSM ) product platform and a  Rationally Selected Microbiota  ( RSM ) product platform based on microbes grown in pure culture. Finch’s lead program, CP101, is an investigational  FSM  product for prevention of recurrent  C. difficile  infections. Finch’s  RSM  platform employs machine-learning algorithms to mine Finch’s unique clinical datasets, reverse engineering successful clinical experience to identify the key microbes driving patient outcomes. Finch has a strategic partnership with Takeda to develop FIN-524, an investigational  RSM  product for inflammatory bowel disease. Finch is using a rich foundation of clinical data to advance its pipeline, leveraging proof-of-principle results to evaluate target indications and inform the design of this new therapeutic class.

Full-Spectrum Microbiota, FSM, Rationally-Selected Microbiota, RSM, and Human-First Discovery are trademarks of Finch Therapeutics Group, Inc.

View source version on businesswire.com:https://www.businesswire.com/news/home/20190208005039/en/

U.S. Food and Drug Administration (FDA) Grants Fast Track Status to Acurx Pharmaceuticals for New Investigational Antibiotic for Clostridium difficile Infection

FDA grants Fast Track status to new C difficile antibiotic

The US Food and Drug Administration (FDA) has granted Fast Track designation to a new investigational antibiotic for Clostridioides difficile infection (CDI), according to a press release yesterday from Acurx Pharmaceuticals.

ACX-362E is a novel, narrow-spectrum oral antibiotic based on inhibition of the enzyme DNA polymerase IIIC, which is required for bacterial replication and pathogenesis in C difficile.

The drug is currently being tested in a phase 1 clinical trial. The company expects to launch a phase 2 trial at the end of year.

Under the Fast Track designation, ACX-362E will receive expedited review from the FDA. The agency grants the designation to drugs that treat serious or life-threatening conditions and fulfill an unmet medical need. The CDC has identified C difficile, which sickens nearly 500,000 Americans each year, as an urgent threat.

“If approved, we believe our new antibacterial, ACX-362E, will be an important therapeutic alternative for patients with CDI,” Acurx managing partner Robert DeLuccia said in the press release. “The Fast Track designation will allow Acurx to work more closely with the FDA to bring ACX-362E to physicians and patients as soon as possible.”
Jan 16 Acurx Pharmaceuticals press release

Information Explaining the “Right to Try” Legislation and What It Means To Terminally Ill Patients

Q: What is the Right to Try Act?

A: Right To Try is legislation that allows terminally ill patients to access investigational treatments that have passed basic safety testing (Phase I) with the FDA, but are not yet available on pharmacy shelves.

Q: Why was Right To Try developed?

A: Over 1 million Americans die from a terminal illness every year. Many spend years searching for a potential cure, or struggle in vain to get accepted into a clinical trial. Unfortunately, FDA red tape and government regulations restrict access to promising new treatments, and for those who do get access, it’s often too late.

Q: Is Right To Try law in my state?

A: Right To Try has been signed into law in 38 states and counting: Alabama, Arizona, Arkansas, California, Colorado, Connecticut, Florida, Georgia, Idaho, Illinois, Indiana, Iowa, Kentucky, Louisiana, Maine, Maryland, Michigan, Minnesota, Mississippi, Missouri, Montana, Nevada, New Hampshire, North Carolina, North Dakota, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, South Dakota, Tennessee, Texas, Utah, Virginia, West Virginia, Washington, and Wyoming. If your state is not listed, and you want to bring Right to Try to your state, click here to find out how.

For more information about the “right-to-try” legislation please click on the following link to be redirected:

http://righttotry.org/faq/

The Senate in August 2017 passed by unanimous consent a measure designed to make it easier for terminally ill patients to get access to experimental treatments without oversight from the U.S. Food and Drug Administration (FDA)

The “right-to-try” legislation has been championed by the libertarian Goldwater Institute, which has worked to pass similar legislation in 37+ states.

The federal version, now headed to the House, would bar the government from blocking patients from getting access to medications that have undergone only preliminary testing in humans. Patients first would have to try all other available treatments and be ineligible for clinical trials.

The bill would provide drug companies some legal protection if a treatment results in harm.

……………..

To read the article in its entirety please click on the following link to be redirected:

https://www.denverpost.com/2017/08/03/right-to-try-terminally-ill-patients-experimental-drugs-fda/

 

 

 

 

Learn More About Clostridium difficile (C.diff., C.difficile) infection and Recurrent CDI Clinical Trials In Progress

 

 

 

The C Diff Foundation has implemented a global campaign to raise awareness of Clostridium difficile infection (C.difficile) clinical trials, clinical studies, clinical research and observational studies evaluating interventions for C. difficile prevention, treatments, and environmental safety.

In the USA: Nearly half a million Americans suffer from Clostridium difficile (C. diff.) infections in a single year according to a study released in 2015 by the Centers for Disease Control and Prevention (CDC). Approximately 29,000 patients died within 30 days of the initial diagnosis of C. difficile. Of those, about 15,000 deaths were estimated to be directly attributable to C. difficile infections making C. difficile a very important cause of infectious disease death in the United States.

“Clostridium difficile infections are not only the most common cause of healthcare-acquired infections in the United States but also very common in the community in younger patients who previously were thought to be less susceptible to C. difficile. The rate of recurrent C. difficile infections is increasing tremendously and this increase is higher than the rate of primary C. difficile infections,” stated Sahil Khanna, MD, Assistant Professor of Medicine Division of Gastroenterology and Hepatology, Director of the C. difficile Clinic, Fecal Microbiota Transplantation program and C. difficile related Clinical Trials, Mayo Clinic, Rochester, MN.

Dr. Khanna also added, “It is imperative and important for clinical trials to be done to advance the development of new treatments, new medications, and new ways to prevent and treat Clostridium difficile infections.”

Individuals volunteer to participate in clinical trials in hopes of improving their own health, to access treatments that might not be available otherwise, often because they are new and not yet widely available. They help others by contributing to advances in medicine. There can also be potential risks participating in clinical trials and clinical studies. All of the known risks associated with a particular trial and or study will be discussed during the informed consent process. It will be thoroughly explained in the informed consent document that a volunteer will receive from the research staff prior to participating in any study.

To learn more about clinical research (e.g., Clostridium difficile, C.difficile) visit the U.S. Food and Drug Administration http://www.fda.gov or telephone 1-800-835-4709, The National Institutes of Health (NIH) http://www.nih.gov and ClinicalTrials.gov.

“Clinical trials are vital to improving our knowledge about how best to prevent and treat C. difficile infections. Informing patients of clinical trials is important, and in recent years several clinical trials have led to significant improvements in the treatments available for patients with C. difficile infections,” stated Mark Wilcox, MD, FRCPath, Consultant Microbiologist, Head of Microbiology and Academic Lead of Pathology Leeds Teaching Hospitals, Professor of Medical Microbiology University of Leeds Institute of Biomedical and Clinical Sciences, Lead on Clostridium difficile for Public Health England, UK.

About the U.S. Food and Drug Administration (FDA):
The FDA is responsible for protecting the public health by assuring that foods are safe, wholesome, sanitary and properly labeled; ensuring that human and veterinary drug, and vaccines and other biological products and medical devices intended for human use are safe and effective. FDA’s responsibilities extend to the 50 United States, the District of Columbia, Puerto Rico, Guam, the Virgin Islands, American Samoa, and other U.S. territories and possessions.

About the National Institutes of Health (NIH):
The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency making important discoveries that improve health and save lives.

About ClinicalTrials.gov
ClinicalTrials.gov is a Web-based resource that provides patients, their family members, health care professionals, researchers, and the public with easy access to information on publicly and privately supported clinical studies on a wide range of diseases and conditions.

U.S. Food and Drug Administration Advises Serious Side Effects Associated With Fluoroquinolone Antibacterial Medication

FDAbanner

The Division of Drug Information (DDI)- serving the public by providing information on human drug products and drug product regulation by FDA.


The U.S. Food and Drug Administration is advising that the serious side effects associated with fluoroquinolone antibacterial drugs generally outweigh the benefits for patients with sinusitis, bronchitis, and uncomplicated urinary tract infections who have other treatment options.  For patients with these conditions, fluoroquinolone should be reserved for those who do not have alternative treatment options. 

The new FDA ruling calling for restricted use of fluoroquinolones affects five prescription antibiotics: ciprofloxacin (Cipro), levofloxacin (Levaquin), moxifloxacin (Avelox), ofloxacin (Floxin), and gemifloxacin (Factive). All are also available as generics.

https://cdifffoundation.org/2016/05/05/a-study-provides-data-that-between-2010-and-2011-throughout-u-s-at-least-30-percent-of-antibiotics-unnecessarily-prescribed/

 

ANTIBIOTIC STEWARDSHIP PROGRAM UPDATES:

https://cdifffoundation.org/2016/04/29/antibiotic-stewardship-program-and-updates-from-sources-cdc-pew-charitable-trusts-with-idsa-and-shea-guidelines/

 

For Additional Information Regarding This Topic – Please Visit The Following Consumer Article:

http://www.consumerreports.org/drugs/fluoroquinolones-are-too-risky-for-common-infections/

An FDA safety review has shown that fluoroquinolones when used systemically (i.e. tablets, capsules, and injectable) are associated with disabling and potentially permanent serious side effects that can occur together.  These side effects can involve the tendons, muscles, joints, nerves, and central nervous system. 

As a result, we are requiring the drug labels and Medication Guides for all fluoroquinolone antibacterial drugs to be updated to reflect this new safety information.  We are continuing to investigate safety issues with fluoroquinolones and will update the public with additional information if it becomes available.

Patients should contact your health care professional immediately if you experience any serious side effects while taking your fluoroquinolone medicine.   Some signs and symptoms of serious side effects include tendon, joint and muscle pain, a “pins and needles” tingling or pricking sensation, confusion, and hallucinations.  Patients should talk with your health care professional if you have any questions or concerns.

Health care professionals should stop systemic fluoroquinolone treatment immediately if a patient reports serious side effects, and switch to a non-fluoroquinolone antibacterial drug to complete the patient’s treatment course.  

Fluoroquinolone drugs work by killing or stopping the growth of bacteria that can cause illness.

We previously communicated safety information associated with systemic fluoroquinolone antibacterial drugs in August 2013 and July 2008.  The safety issues described in this Drug Safety Communication were also discussed at an FDA Advisory Committee meeting in November 2015. 

We urge patients and health care professionals to report side effects involving fluoroquinolone antibacterial drugs and other drugs to the FDA MedWatch program, using the information in the “Contact FDA” box at the bottom of the page.

For more information, please visit: Fluoroquinolone.

 

Fecal Transplants (FMT) Treating Clostridium difficile Infections; U.S. Food and Drug Administration (FDA) Seeks Comment on What Investigational New Drug (IND) Requirements To Waive

Fecal Transplants to Treat C. difficile: FDA Seeks Comment on What IND Requirements to Waive

placeholder+image

The US Food and Drug Administration (FDA) on Monday February 29, 2016,announced new draft guidance that aims to further assure that patients infected with the bacterium Clostridium difficile and not responding to standard therapies can access poop transplants, also known as fecal microbiota for transplantation (FMT).

FDA considers FMT an investigational new drug (IND), which requires physicians and scientists to file an IND application if they intend to use the treatment for clinical practice or research.

However, FDA has issued guidance stating that FMT may be used to treat 

C. difficile infection not responsive to standard therapies outside of a clinical trial. 

New Guidance

The latest draft guidance offers new notice that FDA intends to exercise enforcement discretion regarding the IND requirements for the use of FMT to treat C. difficile infection.

As far as what FDA wants to discuss on this new draft guidance, the agency says it’s requesting comments on which IND requirements are appropriate to waive.

In particular, FDA is requesting comments on the requirement for institutional review board review of the use of FMT to treat patients with C. difficile infection not responding to standard therapies when the FMT is provided by a stool bank,” FDA says.

Background

The draft guidance comes as over the past few years, FMT, which basically involves the transfer of a healthy donor stool to the bowel of a patient infected with C. difficile, has emerged as an effective means to treat recurrent forms of the bacterial infections, according to a study in the Journal of Law and Biosciences.

Rachel Sachs, an academic fellow at Harvard University’s Petrie-Flom Center for Health Law Policy, Biotechnology and Bioethics, and an author of that study, explained to Focus that previously FDA said it would regulate FMT like a biologic, but that the decentralized, hospital-based model of FMT envisioned in this new draft guidance more closely resembles the agency’s models for regulating tissue or cord blood products.

Two companies – Rebiotix and Seres Therapeutics – have been granted orphan drug designations for their INDs as FMT treatments for recurrent C. difficile infections, which affect between 85,000 and 110,000 people in the US annually.

And Sachs said she’s under the assumption that once a company gets FDA approval for their FMT product, FDA will revoke its enforcement discretion included in this new guidance.

Guidance Details

FDA said Monday it intends to use this discretion for waiving certain IND requirements, provided that:

  • The licensed health care provider treating the patient obtains consent from the patient or his or her legally authorized representative for the use of FMT products. The consent should include, at a minimum, a statement that the use of FMT products to treat C. difficile is investigational and a discussion of its reasonably foreseeable risks;
  • The FMT product is not obtained from a stool bank; and
  • The stool donor and stool are qualified by screening and testing performed under the direction of the licensed health care provider for the purpose of providing the FMT product for treatment of the patient.

And FDA makes clear that an establishment that collects or prepares FMT products “solely under the direction of licensed health care providers for the purpose of treating their patients (e.g., a hospital laboratory) is not considered to be a stool bank under this guidance.”

Sachs co-authored her article with Carolyn Edelstein, director of policy and global partnerships at OpenBiome, a nonprofit stool bank that sells FMT capsules (recommended dose of 30 capsules plus a safety test capsule costs $535, or stool preparations for delivery by colonoscopy, enema, and EGD/naso-enteric tube are $385 each) after conducting first-in-human evaluations (N=4) and a randomized dose-finding study (N=17).

Edelstein told Focus that the draft “suggests that the FDA is seeking to set up a more tailored regulatory scheme, one that considers stool banking separately from small-scale directed donation. We are in favor of seeing stool banking receive more regulatory oversight. We plan to answer the agency’s request for comments on the elements of a regulatory framework that would lend this oversight to the practice of stool banking without unduly burdening the physicians and healthcare facilities using banked material, and by extension, unduly limiting access to the treatment for their patients.”

FDA also explains that there were “difficulties in interpretation” with previous draft guidance, particularly around the provision that the donor be known either to the patient or to the treating licensed health care provider, noting “the revised approach more accurately reflects our intent to mitigate risk, based on the number of patients exposed to a particular donor or manufacturing practice rather than the risk inherent from any one donor.”

But as new FMTs are likely to hit the market as orphan drugs, the bigger issue at play could be associated with cost. Sachs noted that any FDA-approved treatment, particularly since it’s an orphan product, could be expensive (upwards of thousands of dollars for treatment).

 

Source:

http://www.raps.org/Regulatory-Focus/News/2016/02/29/24428/Fecal-Transplants-to-Treat-C-difficile-FDA-Seeks-Comment-on-What-IND-Requirements-to-Waive/

Merck Nearing FDA Approval For Its Antitoxin (bezlotoxumab) For Prevention Of C. difficile Infection (CDI) Recurrence

Merck  is nearing FDA approval for its Clostridium difficile-fighting antibody, picking up the agency’s priority review designation with the promise of a shortened vetting process.

The FDA accepted Merck’s application for bezlotoxumab and promised to hand down a final decision by July 23, shortening the standard 10-month review to 6 months.

The treatment, licensed from Medarex in 2009, is an antibody designed to block C. difficile toxin B, which damages the gut wall and leads to inflammation that trigger abdominal pain and diarrhea. In Phase III, adding bezlotoxumab to standard of care significantly reduced C. difficile recurrence in high-risk patients after 12 weeks, Merck disclosed in September.

Bezlotoxumab is among the most advanced assets in Merck’s infectious disease pipeline, bolstered by the company’s $9.5 billion buyout of antibiotics specialist Cubist Pharmaceuticals last year. Merck is also at work on therapies for pneumonia, bacterial infection and HIV as it awaits approval for a combination treatment targeting hepatitis C.

 

FULL STATEMENT FROM MERCK:

Merck  known as MSD outside the United States and Canada  January 28, 2016  (today) announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for bezlotoxumab, an investigational antitoxin for prevention of Clostridium difficile (C. difficile) infection recurrence.

The FDA granted Priority Review for bezlotoxumab, with a Prescription Drug User Fee Act (PDUFA) action date of July 23, 2016.

The company also has filed a marketing authorization application for bezlotoxumab with the European Medicines Agency (EMA) that is currently under review.

“Recurrence is a major challenge with C. difficile infection, one of the most common healthcare-associated infections in U.S. hospitals,” said Dr. Roy Baynes, senior vice president of clinical development, Merck Research Laboratories. “Currently, there are no therapies approved for the prevention of C. difficile infection recurrence. As part of Merck’s commitment to the fight against infectious diseases, we look forward to continuing to work with the FDA and EMA to bring forward this novel medicine for appropriate patients.”

The application for bezlotoxumab is based in part on data from the pivota lMODIFY I and MODIFY II clinical trials. Data from these trials were previously presented at the Interscience Conference of Antimicrobial Agents and Chemotherapy (ICAAC) and International Congress of Chemotherapy and Infection (ICC) 2015 joint meeting.

About bezlotoxumab

Bezlotoxumab is an investigational antitoxin given in conjunction with standard of care antibiotics that are used in the treatment of C. difficile infection. It is not an antibiotic. Bezlotoxumab is designed to neutralize C. difficile toxin B, a toxin that can damage the gut wall and cause inflammation, leading to C. difficile-associated diarrhea.

About Merck

Today’s Merck is a global health care leader working to help the world be well. Merck is known as MSD outside the United States and Canada. Through our prescription medicines, vaccines, biologic therapies and animal health products, we work with customers and operate in more than 140 countries to deliver innovative health solutions. We also demonstrate our commitment to increasing access to health care through far-reaching policies, programs and partnerships. For more information, visitwww.merck.com and connect with us on TwitterFacebookYouTube and LinkedIn.

Forward-Looking Statement of Merck & Co. Inc., Kenilworth, NJ, USA

This news release of Merck & Co., Inc., Kenilworth, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline products that the products will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s 2014 Annual Report on Form 10-K and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov).

To read this article in its entirety please click on the link below:

http://www.fiercebiotech.com/press-releases/merck-announces-fda-acceptance-biologics-license-application-bezlotoxumab-i