Biovitrum | Date: 2015-04-29
The invention relates to nitisinone (2-[2-nitro-4-(trifluoromethyl)benzoyl]-1,3-cyclohexanedione) for use in the treatment of alkaptonuria, wherein nitisinone is administered in a dose of at least 4 mg per day. The invention also relates to a pharmaceutical composition comprising nitisinone for use in the treatment of alkaptonuria, wherein nitisinone is administered in a dose of at least 4 mg per day.
Biovitrum | Date: 2015-04-14
The present invention relates to a syringe having a barrel, a plunger arranged to travel within said barrel and a plunger rod for driving the plunger in a bidirectional manner. The plunger rod is provided with a head at a distal end thereof and a plurality of protruding finger grip enhancing means axially spaced along a length of the plunger rod.
Agency: Cordis | Branch: FP7 | Program: CP-FP | Phase: HEALTH.2012.2.4.4-1 | Award Amount: 11.00M | Year: 2012
DevelopAKUre is a proposal to fund the clinical development of an orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). AKU is a genetic deficiency of homogentisic acid dioxygenase, causing high levels of homogentisic acid (HGA). Oxidation of HGA to pigment polymer, termed ochronosis, alters connective tissues. This leads to multisystemic damage dominated by premature severe arthritis. Currently, multiple arthroplasty is inevitable since AKU is incurable and there is no effective palliative therapy. No data exists regarding the presence or absence of ochronosis before age 30 years. Hence, it is unknown whether treatment is necessary before then. A potential HGA-lowering therapy with nitisinone is available, but lacks outcome data. Thanks to our existing successful fundamental and clinical research (cell models, animal models, natural history studies), we are now ready for the final stage of clinical development of nitisinone for AKU in order to overcome these challenges. This will involve a dose finding study, a phase 3 clinical trial to prove efficacy, and a cross-sectional study in children and young adults to determine when to start treatment. The results of DevelopAKUre will allow us to make the case to the European Medicines Agency for marketing authorisation of nitisinone for AKU, thereby contributing to the goal of the International Rare Diseases Research Consortium of 200 new therapies by 2020. Our consortium has worked together for five years already. It includes Liverpool Universitys AKU Research Team as the lead applicant, the AKU Society UK patient group for dissemination and patient recruitment, three SMEs (Denmark, Netherlands) for biomarker analysis and clinical trial coordination, an industry partner (Sweden) supplying the drug and regulatory support, three universities (UK, Italy, Slovakia) for the analysis of data, and three clinical trial centres (UK, France, Slovakia) to reach required numbers.
Biovitrum | Date: 2015-07-22
Disclosed herein are a modified sulfamidase, a composition comprising a modified sulfamidase, as well as methods for preparing a modified sulfamidase and therapeutic use of such a sulfamidase. In particular, the present disclosure relates to a modified sulfamidase comprising substantially no epitopes for glycan recognition receptors, thereby enabling transportation of said sulfamidase across the blood brain barrier of a mammal, wherein said sulfamidase has catalytic activity in the brain of said mammal.
Biovitrum | Date: 2013-02-19
The present invention relates to C5 binding polypeptides, comprising a C5 binding motif, BM, which motif consists of an amino acid sequence selected from i) EX_(2)X_(3)X_(4)A X_(6)X_(7)EID X_(11)LPNL X_(16)X_(17)X_(18)QW X_(21)AFIX_(23)X_(26)LX_(28)D, and ii) an amino acid sequence which has at least 86% identity to the sequence defined in i), wherein the polypeptide binds to C5. The present invention moreover relates to C5 binding polypeptides for use in therapy, such as for use in treatment of a C5 related condition, and to methods of treatments.
Biovitrum | Date: 2015-03-05
The invention concerns a liquid pharmaceutical formulation suitable for oral administration, comprising a suspension of an effective amount of micronized 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (nitisinone); and citric acid buffer having a pH in the range of 2.5 to 3.5. The formulation is useful in the treatment of disorders and diseases in which inhibition of 4-hydroxyphenylpyruvate dioxygenase (HPPD) is desirable, e.g. in hereditary tyrosinaemia type I.
Biovitrum | Date: 2015-10-29
The present invention relates to pharmaceutical compositions comprising anakinra as an active compound in the absence of sodium citrate. The said pharmaceutical compositions are useful for the treatment of IL-1 mediated disorders and for decreasing nociceptive pain during such treatment.
Biovitrum | Date: 2014-08-28
The invention relates to a polypeptide capable of binding human complement component 5 (C5), said polypeptide comprising the amino acid sequence wherein [BM] is a C5 binding motif; [L2] is an interconnecting loop; X_(42 )is selected from A and S; X_(43 )is selected from N and E; X_(46 )is selected from A, S and C; X_(52 )is selected from E, N and S; X_(53 )is selected from D, E and S, provided that X_(53 )is not D when X_(52 )is N; and X_(54 )is selected from A and S.
Biovitrum | Date: 2015-03-12
The present invention relates to a method to increase the growth velocity of a human infant, said method comprising the enteral administration to said infant of recombinant human bile-salt-stimulated lipase (rhBSSL). Such method has particular utility for underweight or preterm human infants, particular those in medical need of increasing their growth velocity. The invention also relates to compositions, including infant feeds, kits, packaged-pharmaceutical-products and pharmaceutical compositions, and also to methods to prepare infant feeds. In another aspect, the present invention relates to methods to: (X) protect the small bowel mucosa of a human infant from damage; to (Y) protect an immature intestinal epithelium of a human infant from the deleterious effects of incompletely digested and/or excess fat and/or lipid; and/or to (Z) limit accumulation of incompletely digested and/or excess fat and/or lipid in the ileum of a human infant; said methods in each case comprising the step of enteral administration of rhBSSL.
Biovitrum | Date: 2015-03-12
The present invention relates to a method to increase the absorption by a human infant of at least one unsaturated fatty acid, said method comprising the enteral administration to said infant of recombinant human bile-salt-stimulated lipase (rhBSSL). In another aspect the invention also relates to a method to improve the visual and/or cognitive development of a human infant, said method comprising the enteral administration to said infant of rhBSSL. Such methods have particular utility for preterm human infants, particular those in medical need of increasing their absorption of or availability to such unsaturated fatty acids. In further aspects, the invention relates to kits, packaged-pharmaceutical-products, recombinant human bile-salt-stimulated lipase and pharmaceutical compositions, in each case useful for increasing the absorption by a human infant of at least one unsaturated fatty acid, or for increasing the visual and/or cognitive development of a human infant.