How should pharma approach patient collaboration in the 2020s?
From the growing range of patient entities through to new possibilities stemming from technology and patient-generated data, pharma has more and more ways to innovate when it comes to collaborating with patients.
Now pharma needs to identify the ones that will move the dial commercially. “Clearly there is a shift from patient engagement as a nice-to-have, feel-good activity, to something which is more strategic,” says Vanessa Pott, Director, Patient Advocacy& Strategic Partnerships at Merck. “It’s really important for us to stay abreast of all the external trends.” To stay ahead of the curve in the ...
Source: EyeForPharma - April 1, 2020 Category: Pharmaceuticals Authors: Lucy Fulford Source Type: news
EMA Panel Backs Zolgensma Gene Therapy for SMA EMA Panel Backs Zolgensma Gene Therapy for SMA
Zolgensma is a gene therapy that addresses the genetic root cause of spinal muscular atrophy by replacing the defective or missing SMN1 gene to halt disease progression.International Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - March 28, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news
Novartis wins key European recommendation for gene therapy Zolgensma
Swiss drugmaker Novartis on Friday won a key European recommendation for its gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months. (Source: Reuters: Health)
Source: Reuters: Health - March 27, 2020 Category: Consumer Health News Tags: healthNews Source Type: news
New Financing Could Help BillionToOne Expand Product Offerings
BillionToOne wants to expand its diagnostic offerings and its most recent financing could give it a push in that direction. The Menlo Park, CA-based company has raised $15 million in a follow-on to its series A round.
With this financing, the company BillionToOne has brought in more than $32.5 million. The private company noted previous investors, Hummingbird Ventures and NeoTribe Ventures led the investment round with participation from Y Combinator, Libertus Capital, Pacific 8 Ventures, Civilization Ventures, 500 Startups Istanbul, and HOF Capital.
BillionToOne said it will use the proceeds to support the commercial expa...
Source: MDDI - March 11, 2020 Category: Medical Devices Authors: Omar Ford Tags: IVD Business Source Type: news
Roche ’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Basel, 6 February 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32)[1]was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM),[2] a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for ris...
Source: Roche Investor Update - February 6, 2020 Category: Pharmaceuticals Source Type: news
Roche ’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Basel, 06 February 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32)1 was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM),2 a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for risdi...
Source: Roche Media News - February 6, 2020 Category: Pharmaceuticals Source Type: news
Reminder: Invitation to Roche ’s live audio webcast on efficacy and safety of risdiplam in patients with type 2/3 spinal muscular atrophy presented at SMA Europe 2020
(Source: Roche Investor Update)
Source: Roche Investor Update - January 31, 2020 Category: Pharmaceuticals Source Type: news
Roche's SMA drug trial win bolsters $2 billion sales prospects
Roche's spinal muscular atrophy (SMA) drug risdiplam hit another clinical trial target, the Swiss drugmaker said on Thursday, which analysts said bolsters its prospects of reaching $2 billion in annual sales. (Source: Reuters: Health)
Source: Reuters: Health - January 23, 2020 Category: Consumer Health News Tags: healthNews Source Type: news
Roche ’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy
Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was consistent with its known safety profile and no new saf...
Source: Roche Media News - January 23, 2020 Category: Pharmaceuticals Source Type: news
Roche ’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy
Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was consistent with its known safety profile and no new saf...
Source: Roche Investor Update - January 23, 2020 Category: Pharmaceuticals Source Type: news
Invitation to Roche ’s live audio webcast on efficacy and safety of risdiplam in patients with type 2/3 spinal muscular atrophy presented at SMA Europe 2020
(Source: Roche Investor Update)
Source: Roche Investor Update - January 17, 2020 Category: Pharmaceuticals Source Type: news
Roche aims to 'underwhelm' on SMA drug price to challenge rivals
Swiss drugmaker Roche plans to price its oral spinal muscular atrophy drug (SMA) risdiplam aggressively to challenge two of the world's most expensive medicines, Biogen's Spinraza and Novartis's gene therapy Zolgensma. (Source: Reuters: Health)
Source: Reuters: Health - January 14, 2020 Category: Consumer Health News Tags: healthNews Source Type: news
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community. (Source: The Scientist)
Source: The Scientist - January 7, 2020 Category: Science Tags: News & Opinion Source Type: news
Spinraza funding recommended for spinal muscular atrophy in NZ
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - December 31, 2019 Category: Drugs & Pharmacology Source Type: news
Novartis in talks with patients upset about lottery-like gene therapy giveaway
Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease. (Source: Reuters: Health)
Source: Reuters: Health - December 20, 2019 Category: Consumer Health News Tags: healthNews Source Type: news
