Tags : Pediatric Patients

CSL Behring’s Haegarda (C1 Esterase Inhibitor) Receives the US FDA’s

Shots:   The approval is based on P-lll COMPACT pivotal study and OLE study assessing Haegarda (C1 Esterase Inhibitor, SC) vs PBO in patients aged ≤17yrs. with symptomatic HAE.   Results of COMPACT pivotal study: 95% reduction in the number of HAE attacks, 99% reduction in the use of rescue medication. COMPACT OLE results: all patients […]Read More

AbbVie Reports the US FDA’s Acceptance of BOTOX’s sBLA for

Shots: The US FDA has accepted the Allergan’s sBLA to expand the BOTOX prescribing information for treating detrusor (bladder muscle) overactivity associated with an underlying neurologic condition in pediatric patients aged 5 -17yrs who have an inadequate response to or are intolerant of, or for any reason unwilling to continue anticholinergic medication The sBLA is […]Read More

Gilead to Initiate P-II/III Study Evaluating Remdesivir in Pediatric Patients

Shots: Gilead will begin enrollment in P-II/III clinical trial assessing the safety, tolerability, PK and efficacy of remdesivir in ~50 pediatric patients with moderate-to-severe COVID-19, including newborns through adolescents The trial will be conducted at 30+ sites in the US and EU. Remdesivir is available to pediatric patients with severe COVID-19 under our compassionate use […]Read More

Tolmar’s Fensolvi Received the US FDA’s Approval to Treat Pediatric

Shots: The approval is based on P-III study which involves assessing of leuprolide acetate (LA,45 mg) in 64 children with central (gonadotropin-dependent) precocious puberty evaluating its safety and pharmacokinetics The study achieved its 1E i.e. 87% of children achieved a serum luteinizing hormone concentration of <4 IU/L at six months post injection with most common […]Read More

Eli Lilly’s Taltz (ixekizumab) Receives the US FDA’s Approval for

Shots: The approval is based on P-III study assessing Taltz (20mg for <25kg/40mg for 25-50kg/80 mg for >50 kg @12wks., with 40/80/160mg starting doses, respectively) vs PBO in 171 patients aged 6-18 yrs. with mod. to sev. PsO who are candidates for systemic therapy or phototherapy The P-III study results: patients achieving PASI 75 (89% […]Read More

Sanofi Reports Results of Olipudase alfa in Two Studies for

Shots: The P-II/III study involves assessing olipudase alfa (up to 3mg/kg, IV, q2w) vs PBO in 36 adult patients with ASMD for 52wks. demonstrating reduction in spleen volume (39.5% vs 0.5%); reduction in SRS (8.0 vs 9.3) The P-II study involves assessing of olipudase alfa (up to 3mg/kg, IV, q2w) in 20 pediatric patients aged […]Read More

Janssen Receives EC’s Approval for the Expanded Use of Stelara

Shots: The EC’s approval is based on P-III CADMUS Jr study assessing Stelara (SC) in 44 pediatric patients with mod. to sev. PsO aged 6–11 yrs. from 9 countries. The study follows its prior P-III CADMUS study The P-III CADMUS Jr study results: @12wks. improvement in psoriasis and HrQOL; patients achieved PGA 0/1 (77.3%); PASI […]Read More

Allergan’s BOTOX (onabotulinumtoxinA) Receives the US FDA’s Approval for Pediatric

Shots: The FDA’s approval is based on P-III study assessing the BOTOX in 300+ pediatric patients aged 2-17yrs. being treated for lower limb spasticity. The study included patients with cerebral palsy, but the approved indication excludes lower limb spasticity caused by cerebral palsy due to marketing exclusivity by another company  The approval marks the 11th […]Read More

Roche’s Rituxan (rituximab) Receives the US FDA’s Approval for Two

Shots: The FDA’s approval is based on P-IIa PePRS study assessing MabThera/Rituxan (IV) + glucocorticoids in 25 pediatric patients with active granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) aged 6-17yrs. The P-IIa PePRS study results: the achievement of PVAS remission @6mos. (56%); @12mos. (92%); @18mos. (100%); safety profile in pediatric patients with GPA & […]Read More