Posts in category: Regulatory News

Hillstream BioPharma Inc. (“Hillstream”) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to salinomycin based on nonclinical pharmacology data from HSB-1216 (QUATRAMER salinomycin), a new chemical entity for the treatment of small cell lung cancer. The ODD designation provides seven years of market exclusivity post approval for the HSB-1216.

“We are very excited and pleased that HSB-1216 received orphan designation from the FDA,” stated Randy Milby, Hillstream’s Chief Executive Officer. “Now is an exciting time for the development of medicines for small cell lung cancer. We believe treatment with HSB-1216 may represent a complementary approach to other potential therapies by inhibiting cancer stem cells.”

About HSB-1216 (QUATRAMER Salinomycin)
HSB-1216 is a novel formulation of salinomycin, a potent cancer stem cell inhibitor, encapsulated within QUATRAMER delivery technology. Salinomycin kills cancer stem cells by ferroptosis when iron is sequestered in the lysosomes. Salinomycin was identified as the most potent cancer stem cell (CSC) inhibitor known to-date by who or include reference. CSC represent an elusive population of tumor stem cells that are believed to reproduce and sustain cancer. HSB-1216 has shown strong effect on eliminating CSCs. The QUATRAMER salinomycin concentrates within the tumor and is a highly efficacious inhibitor of lung cancer cells. The encapsulation within the QUATRAMER enables may increase tolerability to salinomycin.

About Small Cell Lung Cancer
Lung cancer is a leading cause of cancer-related mortality in the United States with small cell lung cancer (SCLC) accounting for about 10%-15% of all lung cancer diagnoses and has a five-year survival rate of 6% all Surveillance, Epidemiology and End Results (SEER) stages combined.

About Orphan Drug Designation
The FDA, through its Office of Orphan Products Development, grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides a drug developer with certain benefits and incentives, including seven-year period of US market exclusivity from the date of marketing authorization, waiver of FDA user fees, and tax credits for clinical research.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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The US Food and Drug Administration (FDA) on Thursday issued draft guidance on demonstrating “substantial evidence” of effectiveness for drugs and biologics, expanding on decades old guidance issued to address statutory changes brought on by the Food and Drug Administration Modernization Act of 1997 (FDAMA).

Specifically, the 18-page draft guidance builds on FDA’s 1998 guidance Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products, which detailed FDA’s expectations for evidence to support the approval of new drugs and biologics or applications for supplemental indications in light of FDAMA’s clarification that a single adequate and well-controlled clinical trial and confirmatory evidence can be used to support approval.

While FDA says its “standard for demonstrating effectiveness has not changed” since the 1998 guidance, the agency says that new guidance is necessary to keep up with scientific advancements and changes in drug development over the last two decades.

“As our scientific knowledge advances, and drugs and biologics with novel mechanisms are being developed to treat conditions that were previously without treatments, there is a need for additional guidance on the flexibility in the amount and type of evidence needed to establish their effectiveness,” said FDA Principal Deputy Commissioner Amy Abernethy.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Seattle Genetics on Thursday won U.S. approval for its second drug, eight years after joining the ranks of commercial biotech companies with its blood cancer medicine Adcetris. 

Cleared to treat advanced bladder cancer, Padcev was developed in collaboration with the Japanese drugmaker Astellas. The two companies will jointly sell the drug in the U.S., splitting profits and costs equally. 

For Seattle Genetics, the approval caps a year in which the Washington-based biotech’s market value has more than doubled, to approximately $20 billion. Investors’ enthusiasm is rooted in the company’s expanding pipeline, which, in addition to Padcev, includes a breast cancer drug that Seattle Genetics plans to file for approval in the first three months of next year. 

The biotech plans to put another eight drugs into clinical testing over the next two years, an ambitious plan that company CEO Clay Siegall describes as evidence of Seattle Genetics’ evolution beyond Adcetris. 

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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The Food and Drug Administration on Monday approved AstraZeneca and Merck & Co.’s Lynparza for use in treating pancreatic cancer. The oral pill can now be prescribed for a small share of patients with a specific mutation that also increases the risk of breast and ovarian cancer.

Approval was based on a study that found the drug, when used after chemotherapy, kept patients from dying or having their disease progress for almost twice as long as patients taking a placebo. No overall survival benefit was initially observed, however, tempering the significance of the trial’s success.

National treatment guidelines were revised in July 2019 to recommend use of Lynparza in this patient group. An FDA advisory committee voted 7-5 in favor of approval earlier in December.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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–Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of aPAP.

Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).¹

The Molgradex Breakthrough Therapy designation is based on data from IMPALA, a pivotal Phase 3 clinical study evaluating Molgradex for the treatment of aPAP. Data from the study was recently presented in an oral session at the 2019 European Respiratory Society (ERS) International Congress in Madrid, Spain.

“We are pleased that the FDA recognized Molgradex as a breakthrough therapy for the treatment of aPAP, a debilitating rare lung disease with no approved pharmacologic treatment options,” said Rob Neville, Chief Executive Officer, Savara. “We believe this designation reflects the significance of Molgradex as an investigational product that, based on the IMPALA study, has been demonstrated to improve patient outcomes. Breakthrough designation is designed to provide increased collaboration and more frequent dialogue with the FDA and is an important milestone as we work to determine the best path forward for this product.”

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Molecular Partners AG (SIX:MOLN), a clinical-stage biotech company pioneering the use of DARPin therapeutics to treat serious diseases, announces the receipt of Orphan Drug Designation by the US Food and Drug Administration (FDA) for its novel therapeutic, MP0250, for the treatment of Multiple Myeloma.

MP0250 is a first-in-class, tri-specific multi-DARPin drug candidate neutralizing VEGF-A and HGF and is binding to human serum albumin to increase plasma half-life. The unique mechanism of action of MP0250 represents a new approach to targeting the tumor microenvironment and increase patients’ responses to already approved therapies for multiple myeloma, potentially even after progression.

The mission of the FDA’s Office of Orphan Products Development (OOPD) is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. In fulfilling that task, the OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan drug designation provides incentives for sponsors to develop products for rare diseases. These incentives may include a partial tax credit for certain clinical trial expenditures, the waiver of certain FDA user fees, and potential eligibility for seven years of orphan drug marketing exclusivity, if approved.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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OBI Pharma, Inc., a Taiwan biopharma company (TPEx: 4174), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OBI-999 for the treatment of Pancreatic Cancer. OBI-999 is a first-in-class antibody-drug conjugate targeting Globo H, a glycolipid antigen.

A Phase 1/2 clinical trial of OBI-999 has commenced enrollment at the University of Texas M.D. Anderson Cancer Center, with Dr. Apostolia M. Tsimberidou as the Principal Investigator, in patients with locally advanced or metastatic solid tumors, including Pancreatic, Gastric, Colorectal and Esophageal Cancers (ClinicalTrials.gov Identifier: NCT04084366). The objective of the trial is to verify the safety and preliminary efficacy profile of OBI-999 in these patient populations.

Tillman Pearce, MD, CMO, OBI Pharma noted, “We are very excited about the potential value that OBI-999 may provide to patients with pancreatic cancer given both the high potency we have observed using OBI-999 in pancreatic cancer xenograft models and because many pancreas cancers highly overexpress Globo H, the glycolipid target of OBI-999, using the validated IHC assay that will be available for selecting patients for the Phase 2 portion of this first-in-human clinical trial.”

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Advicenne (Paris:ADVIC) (BSE:ADVIC) (Euronext : ADVIC – FR0013296746), a specialty pharmaceutical company focused on the development and commercialization of therapeutic products for rare kidney disease, today announced that its lead drug candidate, ADV7103, has been granted orphan drug designation by the European Medicines Agency (EMA) for the treatment of cystinuria in Europe.

“The EMA’s positive opinion regarding orphan drug designation for ADV7103 is of significant strategic importance to the development of this product,”comments Dr. Luc-André Granier, Co-Founder and CEO of Advicenne. “This designation entails important advantages relating to the clinical development of ADV7103, as well as direct access to the centralized procedure for marketing authorization and 10-year marketing exclusivity following authorization.”

Orphan drug designation is granted to medicines in development for the treatment, prevention and diagnosis of serious or debilitating illnesses whose prevalence does not exceed 5 out of 10,000 people in the European Union and for which there exists no satisfactory solutions or alternative treatments.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Merck (NYSE:MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved ERVEBO® (Ebola Zaire Vaccine, Live) (pronounced er-VEE-boh) for the prevention of disease caused by Zaire ebolavirus in individuals 18 years of age and older. The duration of protection conferred by ERVEBO is unknown. ERVEBO does not protect against other species of Ebolavirus or Marburgvirus. Effectiveness of the vaccine when administered concurrently with antiviral medication, immune globulin (IG), and/or blood or plasma transfusions is unknown. Do not administer ERVEBO to individuals with a history of a severe allergic reaction (e.g., anaphylaxis) to any component of the vaccine, including rice protein.

“Approval of this vaccine by the FDA represents another important milestone in the global response to Ebola Virus Disease and stands as a tremendous accomplishment by a unique global partnership,” said Dr. Roger M. Perlmutter president, Merck Research Laboratories. “In acknowledging this event, I wish in particular to recognize the heroic efforts being made by frontline responders to the ongoing outbreak in the Democratic Republic of the Congo. We are proud and honored to play a role in supporting their vital activities, and we remain focused on the important work ahead.”

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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