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Apellis and Sobi Announce First Patient Dosed in Phase 3 VALIANT Study of Pegcetacoplan for IC-MPGN and C3G, Rare Kidney Diseases with High Unmet Medical Need


WALTHAM, Mass. and STOCKHOLM, Sweden, June 07, 2022 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® (STO:SOBI) today announced that the first patient has been dosed in the Phase 3 VALIANT study investigating pegcetacoplan, a targeted C3 therapy, in primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), two rare and debilitating kidney diseases with similar underlying causes and no approved treatment.

“Individuals with IC-MPGN and C3G experience a high burden of disease due to the lack of approved treatments to slow or stop disease progression. These diseases often lead to kidney failure, requiring dialysis or a kidney transplant, imposing further substantial burdens,” said Kristen Hood, MSN, RN, executive director of research engagement at Nephcure Kidney International. “We are excited that Apellis and Sobi are advancing a Phase 3 study of pegcetacoplan in patients aged 12 and older, with primary IC-MPGN or C3G, including those with post-transplant recurrence.”

Uncontrolled activation of the complement cascade, a part of the body’s immune system, is believed to play a critical role in the progression of IC-MPGN and C3G where excessive accumulation of C3 breakdown products in the kidney causes inflammation and organ damage.1-4 It is estimated that 5,000 people in the United States and up to 8,000 in Europe are living with IC-MPGN or C3G,5 and approximately 50% ultimately suffer from kidney failure within five to 10 years of diagnosis.6

Click here to read the full article at FDANews

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 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 unmet medical needs. 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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FDA Grants Sanofi and Sobi Factor VIII Therapy Breakthrough Designation


The FDA granted Sanofi and Sobi’s investigational blood-clotting drug efanesoctocog alfa a Breakthrough Therapy designation for treatment of patients with hemophilia A.

Efanesoctocog alfa is a novel recombinant factor VIII therapy designed to extend protection from bleeds with a once-weekly prophylactic dose.

The breakthrough designation was supported by phase 3 data showing that the treatment helped prevent bleeding in individuals with the rare, life-threatening genetic bleeding disorder over a 52-week period.

The FDA granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.

Click here to read the full article at FDANews

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 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 unmet medical needs. 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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Kymera Therapeutics Receives FDA Orphan Drug Designation for KT-333, a First-in-Class, Investigational STAT3 Degrader for the Treatment of Peripheral T-Cell Lymphoma


WATERTOWN, Mass., June 01, 2022 (GLOBE NEWSWIRE) — Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing targeted protein degradation to deliver novel small molecule protein degrader medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to KT-333 for the treatment of Peripheral T-cell Lymphoma (PTCL). KT-333 is a first-in-class degrader of the transcriptional regulator STAT3. STAT3 activation has been shown to be a key modulator of disease in PTCL, and there are currently no approved therapies for PTCL that target this pathway.

“The Orphan Drug Designation highlights the potential of this first-in-class heterobifunctional degrader to transform the treatment of PTCL by targeting STAT3, a protein that has historically been undruggable,” said Nello Mainolfi, PhD, Co-Founder, President and CEO, Kymera Therapeutics. “We look forward to working with the lymphoma community to rapidly advance KT-333 as a potential treatment for PTCL while we continue to expand clinical investigation of this novel mechanism in other cancers both in the hematological and in the solid tumor space.”

The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

Click here to read the full article at GlobeNewswire

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 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 unmet medical needs. 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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FDA Gives Accelerated Approval to Novartis’ Kymriah for Follicular Lymphoma


The FDA has granted accelerated approval for Novartis’ chimeric antigen receptor (CAR)-T cell drug Kymriah (tisagenlecleucel) for the treatment of patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

The approval was supported by data from a 90-participant clinical trial in which 68 percent of patients experienced a complete response.

Under the terms of the accelerated approval, the company must conduct one or more confirmatory clinical trials to demonstrate the treatment’s benefit.

Kymriah is also approved for patients up to 25 years old who have B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse and for patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.

Click here to read the full article at FDANews

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 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 unmet medical needs. 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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FDA Expands Approval of SMA Drug Evrysdi to Include Infants Under Two Months Old


The FDA has granted an expanded approval for Roche’s oral solution drug Evrysdi (risdiplam) to include pediatric patients under two months old with the rare genetic disorder spinal muscular atrophy (SMA).

The agency based the expanded approval on interim data from a study that enrolled infants aged from birth to six weeks old at first dose who were genetically diagnosed with SMA but not yet symptomatic.

The majority of presymptomatic infants treated with Evrysdi achieved key milestones, including sitting and standing, with half of the participants walking after 12 months of treatment, the company said.

In August 2020, the FDA approved Evrysdi for pediatric and adult SMA patients aged two months and older.

Click here to read the full article at FDANews

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 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 unmet medical needs. 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.

BPG BLOG (54)

Servier Gets Expanded FDA Approval of Tibsovo for IDH1-Mutated AML


The FDA has approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) in combination with Bristol Myers Squibb’s Vidaza (IV formulation of azacitidine) to treat patients 75 years and older with newly diagnosed isocitrate dehydrogenase-1 (IDH1) mutation in acute myeloid leukemia (AML) or who have comorbidities that preclude use of intensive induction chemotherapy.

“People living with acute myeloid leukemia, especially those who are newly diagnosed and are not eligible for intensive chemotherapy, have had few treatment options,” said Susan Pandya, vice president of clinical development and head of cancer metabolism global development oncology & immuno-oncology at Servier.

Tibsovo, an IDH1 inhibitor, was first approved by the FDA in 2018. The expanded approval for IDH1-mutated AML joins the list of other approved indications, including relapsed or refractory AML and previously treated locally advanced or metastatic cholangiocarcinoma.

Click here to read the full article at FDANews

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 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 unmet medical needs. 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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Blue Lake Biotechnology Announces FDA Fast Track Designation for BLB-201 Intranasal RSV Vaccine


ATHENS, Ga. and LOS GATOS, Calif., May 24, 2022 /PRNewswire/ — Blue Lake Biotechnology, Inc., a clinical-stage biopharmaceutical company developing vaccines using a proprietary transformational parainfluenza virus 5 (PIV5)-based vector, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for BLB-201, an intranasal vaccine for the prevention of respiratory syncytial virus (RSV) associated disease in adults over 60 and children under 2 years of age. BLB-201 is based on an attenuated strain of PIV5 (also known as canine parainfluenza virus) and expresses the RSV-F protein. In preclinical studies, BLB-201, delivered intranasally as a single dose, induces serum antibody and mucosal antibody responses as well as cell-mediated immune responses, and is protective in RSV challenge studies conducted in various animal models. Delivered through the nose without injections, the company’s intranasal vaccines have the potential to facilitate delivery of vaccines to broad populations, including pediatric and needle-hesitant groups.

BLB-201 is one of the few RSV vaccines to have received Fast Track designation for development for use in children under 2 years old. “FDA’s granting of Fast Track designation for BLB-201 reflects the urgent need for an RSV vaccine,” said Dr. Biao He, founder and CEO of Blue Lake Biotechnology and its parent company, CyanVac LLC. “It is encouraging that FDA recognizes the potential of our vaccine to address this need for children under 2 years old as well as for older adults.” Dr. He added, “This RSV vaccine candidate expands our intranasal vaccine pipeline, and builds on our experience in developing a clinical stage intranasal COVID-19 vaccine.”

Blue Lake is preparing to initiate a Phase 1 trial to assess the safety and immunogenicity of BLB-201 in healthy volunteers. The trial is led by principal investigator Paul Spearman, MD, Albert B. Sabin Professor and Director of Infectious Diseases at Cincinnati Children’s Hospital Medical Center.

Click here to read the full article at Cision

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 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 unmet medical needs. 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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Regeneron’s Evinacumab Fights Ultra-Rare Form of High Cholesterol in Kids

Regeneron Pharmaceuticals shared positive results from its Phase III trial of a candidate drug for children with homozygous familial hypercholesterolemia (HoFH), a rare, life-threatening condition characterized by elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease (ACVD.

The latest Phase III trial results evaluated Evkeeza to treat HoFH, an inherited disease that is also the most severe form of familial hypercholesterolemia. HoFH affects one in 160,000 to 300,000 people worldwide, and around 1,300 people in the United States. Those diagnosed with the disease, including patients as young as teenagers, are at high risk for premature atherosclerotic disease and life-threatening cardiac events.

Evekeeza (evinacumab) was developed using Regeneron’s VelocImmune technology, which uses a genetically-engineered mouse platform with a humanized immune system to create fully human antibodies. It’s a fully human monoclonal antibody that binds to and blocks ANGPTL3, inhibiting lipoprotein lipase and endothelial lipase and regulating LDL-C and other circulating lipids. 

Click here to read the full article at BioSpace

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 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 unmet medical needs. 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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PTC’s first gene therapy gains European regulator’s backing

The recommendation from the EMA’s human drugs committee is good news for the gene therapy field after a string of negative developments. Clinical and regulatory setbacks have weighed on company valuations, limiting their fundraising options and leading to restructuring and layoffs for a number of gene therapy developers.

PTC’s regulatory decision is the first of several expected in the coming months that, if positive, could help turn investors’ views around. After its European disappointment, Bluebird is expecting Food and Drug Administration decisions on its two gene therapies this year, while closely watched projects from BioMarin Pharmaceutical and UniQure could also be under FDA review soon. Likewise, PTC expects by the end of September to ask the FDA for approval of Upstaza.

PTC’s therapy corrects a mutation that disrupts production of an enzyme called aromatic L-amino acid decarboxylase, which is in turn vital to production of other chemicals needed for nervous system signaling. As with Luxturna and Zolgensma, Upstaza uses an adeno-associated virus to deliver the corrected gene into cells.

Click here to read the full article at Biopharma Dive

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 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 unmet medical needs. 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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Mersana Enters Busy Gastric Cancer Space with Orphan Drug Designation

On Thursday, Mersana Therapeutics announced that the U.S. Food and Drug Administration granted an orphan drug designation to one of its lead assets, XMT-2056, which is intended to treat gastric cancer.

Gastric cancer, also known as stomach cancer, accounts for approximately 1.5% of all new cancers diagnosed in the U.S. each year. Early signs of gastric cancer can include stomach discomfort, indigestion, nausea and being bloated, but the disease is typically not diagnosed until it is more advanced, causing symptoms such as blood in the stool, vomiting, weight loss, jaundice and fluid in the abdomen. Because it is often not diagnosed until advanced stages, treatment can be challenging. 

Mersana is coming to the treatment arena with XMT-2056. The therapeutic is a differentiated antibody that binds to a novel HER2 epitope, a growth-promoter protein found on some malignant cells. Mersana is investigating the therapeutic as both a monotherapy and a combinatorial therapy with other anti-HER2 treatments. A Phase I trial is anticipated to begin in mid-2022, and the therapeutic will be evaluated in gastric, breast and non-small-cell lung cancer. 

Click here to read the full article at BioSpace

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 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 unmet medical needs. 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.