BPG BLOG (7)

Rare Disease Highlight: Buruli Ulcer

Buruli ulcer (BU) is a chronic skin infection caused by Mycobacterium ulcerans (M. ulcerans) resulting in death of body tissue, also referred to as necrosis (Scherr et al., 2018). M. ulcerans is the third most common mycobacterial infection worldwide and is classified as a neglected tropical disease by the World Health Organization (WHO) (O’Brien et al., 2018). Cases ofBU have occurred in at least 33 countries, with the highest concentration of cases in Western Africa (Guarner, 2018). M. ulcerans cases are also found in Australia (Guarner, 2018; Thomas et al., 2014). BU is not endemic to the US, however, rare cases have occurred in travelers returning from endemic regions (Thomas et al., 2014). The exact mode of transmission of BU is unknown, and studies have investigated potential risk factors such as insect bites and injuries occurring near bodies of water contaminated with M. ulcerans. BU is not considered a contagious disease, as there are no known cases of human-to-human transmission (Guarner, 2018; Thomas et al., 2014).

M. ulcerans mycobacteria produce a toxin, mycolactone, which diffuses out of the mycobacteria and accumulates in important cells in the body, such as fibroblasts and macrophages (Guarner, 2018). Fibroblasts contribute to the formation of connective tissue that supports and connects other tissues or organs in the body, while macrophages are critical for immunity and tissue development and repair (NIH, 2022; Watanabe et al., 2019). Thus, mycolactone targets and binds to scaffolding and other proteins, disrupting cellular structure and function (Guarner, 2018). The downstream effects of the mycolactone toxin can lead to BU. BU starts as a lesion, which progresses to extensive skin ulceration. Early stages of the disease are significantly painful, but as the disease progresses, the lesional loss of tissue is painless or with limited pain. This is thought to be a result of mycolactone activating type 2 angiotensin II receptors (Yotsu et al., 2018). These receptors are associated with regulating pain, so activation decreases sensations to the skin (Pulakat et al., 2020; Yotsu et al., 2018). Patients left untreated have a risk of disease progression resulting in high morbidity, including physical deformities from skin loss and permanent disability as a result of the spread of necrotizing skin infection to bones and joints (O’Brien et al., 2018).

Effective therapeutic options for BU are limited in the US and worldwide. Patients diagnosed and treated in early stages of the disease have a good prognosis and treatment outcome. However, patients left untreated have a risk of disease progression resulting in high morbidity and permanent disability (O’Brien et al., 2018). Standard of care treatment for BU consists of a combination of antibiotics and complementary treatments. The combination of rifampicin (RIF, 10 mg/kg once daily) and clarithromycin (CLR, 7.5 mg/kg twice daily) is now the recommended treatment by the WHO (WHO, 2022). Despite the success of the RIF+CLR combination therapies, these regimens still require 2 months of treatment, often resulting in patient or provider nonadherence (Phillips et al., 2020). Considering the severe morbidity of the disease and the limited treatment options for patients with BU, novel therapies are necessary to address the unmet medical need for this patient population.

References

Guarner. (2018). Buruli Ulcer: Review of a Neglected Skin Mycobacterial Disease. Journal of Clinical Microbiology, 56(4), e01507-01517. doi:10.1128/jcm.01507-17

NIH. (2022). Fibroblast. Retrieved 07/11/2022 from https://www.genome.gov/genetics-glossary/Fibroblast#:~:text=A%20fibroblast%20is%20a%20type,the%20structural%20framework%20of%20tissues.

O’Brien, Jeanne, Blasdell, Avumegah, & Athan. (2018). The changing epidemiology worldwide of Mycobacterium ulcerans. Epidemiol Infect, 1-8. doi:10.1017/S0950268818002662

Phillips, Robert, Abass, Thompson, Sarfo, Wilson, Sarpong, Gateau, Chauty, Omollo, Ochieng Otieno, Egondi, Ampadu, Agossadou, Marion, Ganlonon, Wansbrough-Jones, Grosset, Macdonald, Treadwell, Saunderson, Paintsil, Lehman, Frimpong, Sarpong, Saizonou, Tiendrebeogo, Ohene, Stienstra, Asiedu, van der Werf, Osei Mireku, Abotsi, Adu Poku, Asamoah-Frimpong, Osei-Wusu, Sarpong, Konadu, Opoku, Forson, Ndogyele, Ofori, Aboagye, Berko, Amofa, Nsiah, Mensah-Bonsu, Ofori Nyarko, Amoako, Koranteng Tannor, Boakye-Appiah, Dzibordzi Loglo, Sarpong-Duah, Agbavor, Ardent, Yamadjako, Adanmado Gersande, Adeye, Kindjinou, Akpolan, Kiki, Sodjinou, Guegnard, Klis, Velding, Omansen, Ofori-Adjei, Eyangoh, Knell, & Faber. (2020). Rifampicin and clarithromycin (extended release) versus rifampicin and streptomycin for limited Buruli ulcer lesions: a randomised, open-label, non-inferiority phase 3 trial. The Lancet, 395(10232), 1259-1267. doi:10.1016/S0140-6736(20)30047-7

Pulakat, & Sumners. (2020). Angiotensin Type 2 Receptors: Painful, or Not? Front Pharmacol, 11, 571994. doi:10.3389/fphar.2020.571994

Scherr, Bieri, Thomas, Chauffour, Kalia, Schneide, Ruf, Lamelas, Manimekalai, Grüber, Ishii, Suzuki, Tanner, Moraski, Miller, Witschel, Jarlier, Pluschke, & Pethe. (2018). Targeting the Mycobacterium ulcerans cytochrome bc1:aa3 for the treatment of Buruli ulcer. Nature Communications, 9(1), 5370. doi:10.1038/s41467-018-07804-8

Thomas, Bailey, Bhatnagar, Ritter, Emery, Jassim, Hornstra, & George. (2014). Mycobacterium ulcerans infection imported from Australia to Missouri, USA, 2012. Emerg Infect Dis, 20(11), 1876-1879. doi:10.3201/eid2011.131534

Watanabe, Alexander, Misharin, & Budinger. (2019). The role of macrophages in the resolution of inflammation. J Clin Invest, 129(7), 2619-2628. doi:10.1172/jci124615

WHO. (2022). Buruli ulcer (Mycobacterium ulcerans infection). Retrieved 07/01/2022 from https://www.who.int/news-room/fact-sheets/detail/buruli-ulcer-(mycobacterium-ulcerans-infection).

Yotsu, Suzuki, Simmonds, Bedimo, Ablordey, Yeboah-Manu, Phillips, & Asiedu. (2018). Buruli Ulcer: a Review of the Current Knowledge. Curr Trop Med Rep, 5(4), 247-256. doi:10.1007/s40475-018-0166-2

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 a 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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Regulus Therapeutics Announces Receipt of FDA Orphan Drug Designation for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)


SAN DIEGO, June 21, 2022 /PRNewswire/ — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the “Company” or “Regulus”), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).

“Patients living with ADPKD currently have limited treatment options and approximately half of patients develop end-stage renal disease by age 60 requiring dialysis or transplantation,” commented Jay Hagan, CEO of Regulus Therapeutics. “ADPKD is a disease of high unmet need affecting nearly 160,000 Americans. We look forward to advancing RGLS8429 through the clinic with the goal of improving future treatment options for patients in need.”

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan designation status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

Click here to read the full article at Cision PR Newswire

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 (5)

Phanes Therapeutics’ PT217 receives Orphan Drug Designation for small cell lung cancer from the FDA


SAN DIEGO, June 21, 2022 /PRNewswire/ — Phanes Therapeutics, Inc. (Phanes), an emerging leader in innovative discovery research and clinical development in oncology, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PT217 for the treatment of small cell lung cancer (SCLC).

PT217 is a first-in-class bispecific antibody targeting Delta-like ligand 3 (DLL3) and cluster of differentiation 47 (CD47) being developed for patients with SCLC and other neuroendocrine cancers.  SCLC is an aggressive pulmonary carcinoma hallmarked by high early mortality rates and significant morbidities throughout the disease’s progression. The 1-year survival of patients with SCLC is only 32.9%, with only 10.7% of patients surviving 3 years.

“PT217 has the potential to be a transformative treatment option for SCLC patients whose initial response to chemotherapy is short-lived and inevitably becomes resistant to chemotherapeutic agents” said Dr. Ming Wang, Founder and CEO of Phanes Therapeutics. “We have built a strong pipeline in immuno-oncology by leveraging our proprietary technology platforms and expect to file an IND for PT217 by the third quarter of this year. This orphan drug designation follows two recent IND clearances for our PT199, an anti-CD73 monoclonal antibody and PT886, an anti-Claudin 18.2/anti-CD47 bispecific antibody, programs which we are progressing into the clinic.”

Click here to read the full article at Cision PR Newswire

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 (65)

LG Chem’s New Drug for Obesity Receives Additional Orphan Drug Designation by the US FDA


LG Chem announced on 16th that the FDA recently granted ODD to ‘LB54640’ for the treatment of ‘POMC (Proopiomelanocortin) deficiency.’

‘LB54640’ also received ODD back in September of 2020 by the FDA for ‘LEPR (leptin receptor) deficiency’.

The FDA operates the ODD system to encourage and support the development of rare and difficult-to-treat disease drugs with prevalence rates of less than 200,000 people. It is estimated that there are about 120,000 people suffering from rare genetic obesity in the US.

With this designation, LG Chem will receive benefits such as exclusive sales rights in the US market for seven years, subsidies and tax cuts for clinical trial expenses in the US, and preliminary consulting support related to development.

Click here to read the full article at Business Wire

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 (64)

Rare Disease Highlight: Congenital disorder of glycosylation type IIc (CDGIIc)

Leukocyte Adhesion Deficiencies (LAD) are autosomal recessive immunodeficiencies characterized by severe and recurrent bacterial infections, impaired wound healing, and neutrophilia (Cagdas et al., 2014). Congenital disorder of glycosylation type IIc (CDGIIc) is an ultra-rare LAD in which patients exhibit these general LAD phenotypes, but are sometimes further characterized by two additional potential symptoms including moderate to severe psychomotor retardation and mild dysmorphism (OMIM, 2014). In CDGIIc patients, the body is unable to sufficiently add fucose sugars to the surface of certain cells (hypo-fucosylation), which causes leukocytosis, persistent and recurrent infections, severely reduced selectin activity, Bombay blood group, hypotonia, poor feeding, short stature, coarse facial features and neurological deficits. This is mainly because the leukocytes in the body are unable to adhere to cell surfaces which would normally be marked with fucose sugars to fight off infections (Marquardt et al., 1999). In some cases, patients died because of clinical manifestations of the disease (confirmed after death), demonstrating the severe and life-threatening nature of CDGIIc (Cagdas et al., 2014; Karsan et al., 1998).

Immune defects are the direct result of a lack of cluster of differentiation (CD) 15, a fucose-containing, cell surface glycoprotein that serves as a ligand for certain cell adhesion molecules called E- and P‑selectins (Frydman et al., 1996). In addition, CDGIIc patients’ red blood cells lack the H antigen, a fucosylated glycoprotein, which is the precursor molecule of the A, B, and O blood groups, and consequently manifests as the Bombay blood type. Recurrent bacterial infections occur in almost all patients during the first years of life, although periodontitis has been reported at later ages (A. Etzioni et al., 1992). Biochemical analysis confirms global hypo-fucosylation of proteoglycans, suggesting an inborn error of fucose metabolism. CDGIIc is a serious and potentially life-threatening disease, particularly for infants and young children (Dauber et al., 2014; A. Etzioni et al., 1992; Amos Etzioni et al., 2002; Frydman et al., 1992; Karsan et al., 1998; Marquardt et al., 1999; Sturla et al., 1998). The onset of clinical symptoms is typically during infancy and almost always within the first year of life. Without medical intervention, clinical manifestations of CDGIIc, in most cases, persist through development or result in patient mortality during early childhood (Dauber et al., 2014; A. Etzioni et al., 1992; Amos Etzioni et al., 2002; Frydman et al., 1992; Marquardt et al., 1999; Sturla et al., 1998)

References

Cagdas, D., Yilmaz, M., Kandemir, N., Tezcan, I., Etzioni, A., & Sanal, Ö. (2014). A novel mutation in leukocyte adhesion deficiency type II/CDGIIc. Journal of clinical immunology, 34(8), 1009–1014. doi:10.1007/s10875-014-0091-7

Dauber, A., Ercan, A., Lee, J., James, P., Jacobs, P. P., Ashline, D. J., . . . Sackstein, R. (2014). Congenital disorder of fucosylation type 2c (LADII) presenting with short stature and developmental delay with minimal adhesion defect. Human molecular genetics, 23(11), 2880–2887. doi:10.1093/hmg/ddu001

Etzioni, A., Frydman, M., Pollack, S., Avidor, I., Phillips, M. L., Paulson, J. C., & Gershoni-Baruch, R. (1992). Brief report: Recurrent severe infections caused by a novel leukocyte adhesion deficiency. N Engl.J Med, 327(25), 1789–1792. doi:10.1056/nejm199212173272505

Etzioni, A., Sturla, L., Antonellis, A., Green, E. D., Gershoni-Baruch, R., Berninsone, P. M., . . . Tonetti, M. (2002). Leukocyte adhesion deficiency (LAD) type II/carbohydrate deficient glycoprotein (CDG) IIc founder effect and genotype/phenotype correlation. Am J Med Genet, 110(2), 131–135. doi:10.1002/ajmg.10423

Frydman, M., Etzioni, A., Eidlitz-Markus, T., Avidor, I., Varsano, I., Shechter, Y., . . . Gershoni-Baruch, R. (1992). Rambam-Hasharon syndrome of psychomotor retardation, short stature, defective neutrophil motility, and Bombay phenotype. Am J Med Genet, 44(3), 297–302. doi:10.1002/ajmg.1320440307

Frydman, M., Vardimon, D., Shalev, E., & Orlin, J. B. (1996). Prenatal diagnosis of Rambam-Hasharon syndrome. Prenat Diagn, 16(3), 266-269. doi:10.1002/(sici)1097-0223(199603)16:3<266::Aid-pd845>3.0.Co;2-#

Karsan, A., Cornejo, C. J., Winn, R. K., Schwartz, B. R., Way, W., Lannir, N., . . . Harlan, J. M. (1998). Leukocyte Adhesion Deficiency Type II is a generalized defect of de novo GDP-fucose biosynthesis. Endothelial cell fucosylation is not required for neutrophil rolling on human nonlymphoid endothelium. J Clin Invest, 101(11), 2438–2445. doi:10.1172/jci905

Marquardt, T., Brune, T., Lühn, K., Zimmer, K. P., Körner, C., Fabritz, L., . . . Koch, H. G. (1999). Leukocyte adhesion deficiency II syndrome, a generalized defect in fucose metabolism. J Pediatr, 134(6), 681–688.

OMIM. (2014). Online Mendelian Inheritance in Man: MIM Number: 266265. Retrieved from https://omim.org

Sturla, L., Etzioni, A., Bisso, A., Zanardi, D., Flora, G. d., Silengo, L., . . . Tonetti, M. (1998). Defective intracellular activity of GDP-D-mannose-4,6-dehydratase in leukocyte adhesion deficiency type II syndrome. FEBS Lett, 429(3), 274–278.

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 a 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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ResVita Bio receives Rare Pediatric Disease Designation for RVB-001 as a Treatment for Netherton Syndrome


BERKELEY, Calif., June 9, 2022 /PRNewswire/ — ResVita Bio, a cellular therapies startup, announces that the FDA has granted Rare Pediatric Disease Designation to RVB-001 to treat Netherton Syndrome, a chronic and life-threatening genetic skin disease.

“The physical and emotional challenges of chronic skin diseases are devastating to patients, particularly children” said Amin Zargar, Chief Executive Officer of ResVita Bio. “Newborns with Netherton Syndrome face a severe lifelong disease with few treatment options that offer limited efficacy. We are motivated by the prospect to deliver meaningful improvement in the lives of these patients, and this is a significant step towards the development RVB-001 as a treatment for Netherton Syndrome.”

With the Rare Pediatric Disease Designation, if a new drug application for RVB-001 is approved, ResVita Bio may be eligible to receive a priority review voucher, which can be redeemed for priority review of any subsequent drug marketing application. The voucher can also be sold or transferred.

Click here to read the full article at Cision PR Newswire

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 (4)

Vertex Kidney Disease Drug Scores FDA Breakthrough Therapy Designation


Vertex Pharmaceuticals announced on Wednesday that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to its candidate, inaxaplin (VX-147). Inaxaplin was granted the designation for the treatment of APOL1-mediated focal segment glomerulosclerosis (FSGS).

APOL1-mediated FSGS is a kidney disease in which scar tissue develops on parts of the kidney called glomeruli, which is associated with APOL1 genetic mutations. The APOL1 gene is associated with the innate immune system, and people who have two mutations in the gene are at an increased risk of developing kidney diseases such as FSGS.

This is due to increased toxicity of the APOL1 protein in the kidney, which is the result of inflammatory exposures. Currently, there are no approved treatments for the disease, although treatments such as high-dose steroids can delay the progression to kidney failure.

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.

BPG BLOG (61)

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.