Posts tagged: Unmet need

Acucela Inc. (“Acucela”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596), announced today that the European Medicines Agency (EMA) granted orphan designation to Acucela’s leading drug candidate emixustat hydrochloride (“emixustat”) for the treatment of Stargardt disease. The orphan designation does not apply to other indications for which emixustat is being developed.

To receive an orphan designation from the EMA, the medicine has to be developed for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 within the European Union. Upon receiving the orphan designation, the sponsor will qualify for multiple development incentives which include ten years of market exclusivity after product approval, fee reductions of regulatory activities, access to the centralized marketing authorization procedure, and protocol assistance that provides scientific advice for the orphan designated medicines.

In January 2017, Acucela also received orphan drug designation for emixustat for the treatment of Stargardt disease by the FDA (U.S. Food and Drug Administration). According to the FDA’s Center for Drug Evaluation and Research’s annual report, the number of approved orphan drugs has been increasing year by year, and 58% of the drugs approved by the FDA in 2018 were orphan drugs. Many of them are expected to have peak sales of over US$1 billion.

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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 therapeutics 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.

Biosight Ltd., a pharmaceutical development company focused on the development of novel oncology therapeutics, announced today that the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation to BST-236, an investigational novel antimetabolite, for the treatment of acute myeloid leukemia (AML). BST-236 is being developed for the treatment of hematological malignancies and disorders and is currently in Phase 2b development for front-line treatment of AML.

“We are very pleased to have received Orphan Drug Designation from the FDA for BST-236, our lead therapeutic candidate,” said Ruth Ben Yakar, Ph.D., CEO of Biosight. “The emerging clinical data from a completed Phase 1/2a and an ongoing Phase 2b studies, suggest that BST-236 may provide a superior front-line treatment option for AML patients, especially for patients who are medically unfit to receive standard chemotherapy, and may establish BST-236 as the new core therapeutic in AML and other hematological malignancies and disorders.”

Orphan Drug Designation by the FDA entitles Biosight to seven years of market exclusivity for the use of BST-236 for the treatment of AML, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

About BST-236
BST-236 is a novel antimetabolite, a pro-drug of the chemotherapeutic drug cytarabine. Cytarabine has been the backbone of AML therapy for the past 40 years, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically unfit patients.  BST-236 is designed to enable the delivery of high cytarabine doses to leukemia cells with lower systemic exposure to the free drug and relative sparing of normal tissues. As such, BST-236 may serve as a superior core therapy for AML and other hematological malignancies and disorders, including to medically unfit or older adults.

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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 therapeutics 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.

Celgene Corporation (NASDAQ:CELG) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application for ozanimod for the treatment of people with relapsing forms of multiple sclerosis (RMS) in the United States. The European Medicines Agency (EMA) also accepted for review the Marketing Authorization Application for ozanimod for the treatment of adults with relapsing-remitting multiple sclerosis (RRMS) in the European Union. Ozanimod is an oral, sphingosine 1-phosphate (S1P) receptor modulator that binds with high affinity selectively to S1P subtypes 1 (S1P₁) and 5 (S1P₅). Under the Prescription Drug User Fee Act, the FDA has set its action date as March 25, 2020. A regulatory decision from the EMA is expected in the first half of 2020.

Both applications are based primarily on ozanimod data from the SUNBEAM™ and RADIANCE™ Part B phase 3, multicenter, randomized, double-blind, double-dummy, active-controlled trials.

“The U.S. Food and Drug Administration and European Medicines Agency acceptances of our applications represent a crucial step forward in our efforts to bring ozanimod to people with multiple sclerosis,” said Jay Backstrom, M.D., Chief Medical Officer for Celgene. “We believe that ozanimod has the potential to be an important option early in the treatment of relapsing forms of MS and a best-in-class S1P receptor modulator.”

Ozanimod is an investigational compound that is not approved for any use in any country.

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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 therapeutics 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.

Bluebird Bio has its first ever product approval, getting a green light from the EMA for Zynteglo, its ex vivo gene therapy for the rare blood disorder beta-thalassemia.

The EU regulator has given a conditional approval to Zynteglo, which is based on CD34+ stem cells taken from patients that are genetically modified to contain a working gene for beta-globin, a hemoglobin component that is deficient in thalassemia patients. Patients are given conditioning chemotherapy to clear their bone marrow of cells before the cells are reinfused.

The one-shot therapy – previously known as LentiGlobin – can now be used in the EU to treat patients aged 12 or over with transfusion-dependent thalassemia (TDT), in other words, those who are the most severely affected by the disorder. Bluebird is carrying out a pair of phase 3 trials and a long-term follow-up study to try to confirm its safety and efficacy.

There’s no word yet on pricing, which is a thorny issue for gene therapy developers as they try to balance a high upfront cost – sometimes millions of dollars as with Novartis’ new Zolgensma therapy for rare disease spinal muscular atrophy – with what should be long-term clinical benefits that can save health systems money.

Bluebird has said it will carry out a “continue the country-by-country reimbursement process to help ensure access to Zynteglo for appropriate patients,” adding that it plans to reveal more detail on its pricing plans by the end of next week.

The biotech has previously discussed a payment model based on an upfront fee and payments on instalment over five years contingent on the therapy still working as expected, with a maximum price of $2.1 million proposed. Analysts suggest the price in Europe is likely to be set lower, perhaps around the €1 million mark.

That level of pricing would be in the same ballpark as allogeneic hematopoietic stem cell transplantation (HSCT), which can be attempted in TDT patients but unlike Zynteglo relies on finding a matched donor and also carries significant risks including treatment-related mortality, graft failure, graft versus host disease (GvHD) and opportunistic infections.

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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 therapeutics 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.

Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical-stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced that the European Commission (EC) has granted orphan drug designation (ODD) to PTI-428 for the treatment of cystic fibrosis. PTI-428 is the Company’s proprietary cystic fibrosis transmembrane conductance regulator (CFTR) amplifier that is currently in clinical development. In addition to ODD from the EC, PTI-428 has ODD, Breakthrough Therapy Designation and Fast Track Designation from the U.S. Food and Drug Administration.

ODD in the European Union (EU) is based upon a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) and provides regulatory and financial incentives for companies to develop and market therapies to treat serious disorders affecting no more than five in 10,000 persons in the EU. Companies that obtain ODD benefit from a number of incentives, including ten-year marketing exclusivity in the EU upon approval, as well as eligibility for protocol assistance, reduced fees and access to the EU’s centralized marketing authorization procedure. Currently, over half of the global CF population lives in Europe with limited access to approved CFTR modulator therapies.

“This designation by the EC is further validation of both PTI-428’s potential and PTI’s mission to offer additional disease-modifying treatment options for CF,” said Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics.  “Orphan drug status is granted to development-stage drugs that make a major contribution to patients’ care, either by demonstrating a significant clinical benefit over existing therapies or by providing treatment for patients for whom existing therapies do not work. We look forward to advancing PTI-428 in the clinic later this year, as part of the planned 28-day Phase 2 studies of our proprietary combination CFTR modulator 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 therapeutics 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.

ARV-110, a PROTAC^® protein degrader, has received a fast track designation from the FDA for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC) who have disease progression following ≥2 systemic therapies.

ARV-110 is an orally bioavailable agent which is designed to selectively target and degrade the androgen receptor (AR), is currently being investigated in a phase I trial (NCT03888612). Researchers are evaluating the pharmacokinetics, safety, and tolerability of the agent in this patient population who have already received standard regimens.

“While great strides have been made in the treatment of men with metastatic castration-resistant prostate cancer, current AR-targeted standard-of-care treatments are less effective in patients whose disease includes increased levels of androgen production or mutations in the androgen receptor,” John Houston, PhD, president and chief executive officer of Arvinas, Inc., the developer of ARV-110, in a press release. “We believe, due to its ability to iteratively degrade the AR protein, ARV-110 could represent a meaningful new therapy to improve the lives of patients battling mCRPC, and for whom current therapies are not effective. The Fast Track designation by the FDA recognizes the urgency for improved treatments for these patients.”

Currently available, standard-of-care AR-targeted regimens for patients with mCRPC are said to be less effective in those with disease that has increased levels of androgen production, AR gene or gene enhancer amplification, or ARpoint mutations. Approximately 15% to 25% of patients do not respond to second-generation hormone therapies, such as abiraterone acetate (Zytiga) and enzalutamide (Xtandi), and the majority of patients who do respond will eventually develop resistance.

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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 therapeutics 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.

Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D. “Astellas”) today announced that the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) to update the U.S. product labeling for XOSPATA^® (gilteritinib) to include final analysis data from the ADMIRAL trial. The data demonstrated improvement in Overall Survival in those treated with gilteritinib monotherapy versus salvage chemotherapy in adult patients with relapsed (disease that has returned) or refractory (resistant to treatment) Acute Myeloid Leukemia (AML) with an FMS-like tyrosine kinase 3 (FLT3) mutation.

“The ADMIRAL trial’s Overall Survival (OS) findings are encouraging for patients and families impacted by relapsed/refractory FLT3 mutation-positive AML,” said Alexander Perl, M.D., Abramson Cancer Center, University of Pennsylvania. “The data underscore the importance of single-agent XOSPATA for this patient population that, until recently, had few remaining treatment options.”

Results from the ADMIRAL trial show the median OS for patients who received XOSPATA was 9.3 months compared to 5.6 months for patients who received salvage chemotherapy (Hazard Ratio = 0.64 (95% CI 0.49, 0.83), P=0.0004). The other co-primary endpoints of Complete Remission (CR)/Complete Remission with Partial Hematologic Recovery (CRh) in the XOSPATA arm at the interim analysis was 21% (95% CI: 14.5, 28.8).¹

The safety profile of XOSPATA is based on 319 patients with relapsed or refractory AML in three clinical trials (NCT02421939, NCT02014558, and NCT02181660). Fatal adverse reactions occurred in 2% of patients receiving XOSPATA. These included cardiac arrest (1%) and one case each of differentiation syndrome and pancreatitis. The most frequent (≥5%) nonhematological serious adverse reactions reported in patients were fever (13%), dyspnea (9%), renal impairment (8%), transaminase increased (6%) and noninfectious diarrhea (5%). The most frequent (≥5%) grade ≥3 nonhematological adverse reactions reported in patients were transaminase increased (21%), dyspnea (12%), hypotension (7%), mucositis (7%), myalgia/arthralgia (7%), and fatigue/malaise (6%).

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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 therapeutics 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.

 AveXis, a Novartis company, announced the US Food and Drug Administration (FDA) has approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.

“A diagnosis of SMA is devastating, leaving untreated babies who have the most severe form with painfully short, highly medicalized lives, during which they are unable to lift their heads, sit or roll, have difficulty swallowing and breathing and need 24-hour care,” said Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy at The Abigail Wexner Research Institute of Nationwide Children’s Hospital in Columbus, OH. “In the START clinical trial we conducted with Zolgensma, all children were alive at the conclusion of the study and many were able to sit, roll, crawl, play and some could walk. This level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families battling SMA Type 1. We now have data four years out from the trial, and we see the durability of this gene therapy.”

“The approval of Zolgensma is a testament to the transformational impact gene therapies can have in reimagining the treatment of life-threatening genetic diseases like spinal muscular atrophy,” said Vas Narasimhan, CEO of Novartis. “We believe Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition.”

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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 therapeutics 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.

The Food and Drug Administration (FDA) has granted Breakthrough Status designation to NightWare (NightWare, Inc.), a digital intervention therapy that measures and treats nightmares in patients with post-traumatic stress disorder (PTSD).

NightWare runs on the Apple Watch platform via a smartwatch application. This device is able to create a profile of a patient’s sleep patterns through integrated sensors and machine learning algorithms, typically after a learning period of fewer than 10 days.

At the onset of a nightmare, the patient is aroused from sleep (without awakening) using vibrotactile feedback so that the nightmare is interrupted prior to the patient waking up in distress. The device will not affect the circadian sleep pattern of the patient; if it senses that the patient is being woken by the vibrations, it will vibrate less the next time.

A desktop app, which displays the data collected by the smartwatch app, allows healthcare providers to review individual patient data in order to provide guidance on managing their nightmare disorder.

The Company is currently enrolling patients in randomized clinical trials, one of which will be virtual. Patients interested in participating in a trial can get more information here.

“The FDA’s Breakthrough Status designation will enable us to complete our randomized clinical trials ahead of schedule and give us the opportunity to help improve the lives of veterans and others suffering from PTSD sooner than we anticipated,”  said NightWare CEO and Founder Grady Hannah.

NightWare is a private digital therapeutics company headquartered in Minneapolis, MN with a platform designed to treat nightmare disorder. It is currently seeking FDA clearance for commercial sales as a medical device.

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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 therapeutics 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.

A nine-year-old drug acquisition for Pfizer has paid off with a nod from the U.S. Food and Drug Administration (FDA) for approval of Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), a first-of-its-kind treatment for a rare heart disease that can lead to heart failure.

On Monday, the FDA gave the green light to Pfizer for the two drugs, which are oral formulations of the first-in-class transthyretin stabilizer tafamidis for the treatment of the cardiomyopathy of wild-type (meaning it can occur as people age) or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. The medicines were quickly approved following Pfizer’s New Drug Application filing late last year. The approval marks the first two medicines approved by the FDA to treat ATTR-CM. The drugs have already been pegged as potential blockbusters, with analysts predicting $2 billion in peak sales. The list price for the ATTR-CM treatment, according to an analysis on Seeking Alpha, has been set at $225,000. Tafamidis has been approved in Europe for transthyretin familial amyloid polyneuropathy (TTR-FAP). It is sold there under the brand name Vyndaqel.

Paul Levesque, global president of Pfizer’s rare disease group, called the approval a game changer for patients with this disease. Until the FDA’s approval, Levesque said these patients had no approved medications to treat their illness.

“Pfizer’s purpose is to deliver breakthrough medicines that change patients’ lives. The approvals of Vyndaqel and Vyndamax deliver on this promise for patients with ATTR-CM,” Levesque said in a statement.

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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 therapeutics 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.