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[posting]46745673[/posting]Dieser SA Schreiberling kennt wohl Santhera nicht wenn jemand eine vorzeitige Zulassung verdient dann wohl eher Santhera immerhin war die Zwischenanalyse mit 65 DMD patienten erfolgreich .Sarepta hat versucht anhand einer 12 patienten Studienergebnis die Zulassung zu erlangen bei so einer kleinen anzahl an patienten ist das ergebnis nicht aussagekräftig kein wunder das die FDA abgelehnt hat . Drücken wir also Santhera die daumen ,wenn die Phase 3 daten gut sind dann könnte Santhera als erstes in den DMD Markt eintreten und wahrscheinlich für lange Zeit auch allein dominieren . Sarepta (SRPT) : About Study 201 and Study 202 (Phase IIb Eteplirsen Study) Study 4658-US-201 was conducted at Nationwide Children's Hospital in Columbus, Ohio. Twelve boys meeting the inclusion criteria being between 7 and 13 years of age with appropriate deletions of the dystrophin gene that confirm eligibility for treatment with an exon-51 skipping drug, received double-blind IV infusions of placebo (n=4), 30 mg/kg of eteplirsen (n=4), or 50 mg/kg of eteplirsen once weekly for 24 weeks (n=4). Muscle biopsies for evaluation of dystrophin were obtained at baseline for all subjects, and after 12 weeks for patients in the 50 mg/kg cohort and after 24 weeks for patients in the 30 mg/kg cohort. Two placebo patients were randomized to the 30 mg/kg cohort and two placebo patients were randomized to the 50 mg/kg cohort. This study design allowed Sarepta to investigate the relationship of dose and duration of eteplirsen treatment on the production of dystrophin over the course of the 24-week study. ----------------- Santhera Pharma : At the end of 2012, a total of 65 steroid non-using DMD patients were enrolled in the randomised, placebo-controlled DELOS trial and in April 2013, the company has passed a planned futility analysis for safety and efficacy for the primary respiratory function endpoint. Santhera senior vice president of Development Nick Coppard said that following the successful futility analysis earlier in 2013, the company is pleased to confirm that the DELOS study remains on track to deliver pivotal efficacy data early in the second quarter of 2014. |
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| aus der Diskussion: | Diskussion zu SANTHERA PHARMACEUTICALS HOLDING AG |
| Autor (Datum des Eintrages): | Biohero (01.04.14 20:22:23) |
| Beitrag: | 32 von 14,080 (ID:46748233) |
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