Pharming « Terug naar discussie overzicht

quote:

fanaat schreef op 21 september 2021 12:40:

De notering aan de NQ is een heel dure hobby van de baas. Hij heeft daarvoor zelfs de penningmeester ingeruild. Nu na 9 maanden nog altijd geen bevalling aldaar en naar ik vermoed komt die ook niet want de bevruchting is iets te onhandig en te vlug gegaan. In de euforie van het gebeuren is de baas het een en ander vergeten.
Inderdaad de koers van Pharming is ondertussen met ruim $ 11 dollar gezakt. Mocht de waarde dalen tot $ 4 dan zijn de rapen gaar. Op het damrak blijft dan helemaal niets over van dit fonds.

quote:

Wijze man schreef op 21 september 2021 12:57:

[...]

Je weet toch hoe het werkt, een vaste woon e/o verblijfplaats en een postbus voor het belastingvoordeel.

Zo zie ik de nasdaq notering.

quote:

Ien72 schreef op 21 september 2021 12:46:

In the conference call van 5 aug jl. heeft SdV een toelichting gegeven op het traceren en vaststellen van APDS patienten die in aanmerking komen voor leniolisib. Zie hieronder.
Uit zijn toelichting blijkt dat veel potentiele patienten bekend zijn omdat ze nu behandeld worden voor immonodeficiency. Met een genetische test kan bij deze patienten de ziekte vastgesteld worden. Op dit moment wordt gewerkt aan een APDS netwerk zodat er snel kan worden overgegaan op behandeling na de goedkeuring.
Wereldwijd zijn er naar verwachting ca 1.350 APDS patienten.

the good news is, this diagnosis can be made by commercially available genetic tests. And all these patients are already treated by immunologist because they form this part of the population -- patient population that's called primary immunodeficiency that is treated symptomatically by these immunologists, and are actually waiting for that disease to be formally discovered and confirmed. And then there could be a treatment of Leniolisib in the future for them.

Next slide, please. And because the Leniolisib launch is coming near, we started investing significantly in uncovering APDS, so we're looking at target and targeted patient identification strategy that is actually kicked off in the US and will be rolled out across Europe as well. And you can see here, we build an APDS network, KOL network and referral pathway of prescribers, that are been actively supported by field medical and diagnostic liaisons from our company that were -- that we started to work recently on this.

We use analytics and artificial intelligence to look at the databases and the patient group that is immunology pass and there we have an outreach and education. And we offer free of charge genetic testing to confirm the disease and then in the future offer -- be able to offer these patients treatment for Leniolisib, that is an important step for a company to start the pre-marketing activities for Leniolisib because Leniolisib is a good year – is only a year and a half away potentially from the mark

quote:

Wijze man schreef op 21 september 2021 13:09:

[...]

Nou, weet je, ik heb het e.e.a vluchtig doorgenomen, dus wat ik nu schrijf is een beetje klok en klepel, maar je kan alleen ADR,s noteren als je een andere notering hebt, in dit geval Nederland, en nu heb ik ook gelezen dat je dan je core aandelen kan inwisselen via een bemiddelaar, bank of broker tegen ADR,s, in dat geval wordt Pharming daar niet veel wijzer van op financieel gebied, maar het is wel een mooie cover om stukje bij beetje van de beurs in Amsterdam te gaan.

quote:

Sharen schreef op 21 september 2021 13:12:

[...]
Klopt, en dat is NU al beschikbaar :

www.invitae.com/en/navigateAPDS/

quote:

DE MONITOR - Dé Pharming Expert schreef op 21 september 2021 13:11:

Oral Orladeyo Now Available in UK to Treat HAE Swelling Attacks

by Marisa Wexler MS | September 20, 2021

oral Orladeyo now available in UK | Angioedema News | woman with megaphone announcement illustration
Orladeyo (berotralstat), a daily oral therapy to prevent swelling attacks in people with hereditary angioedema (HAE), is now available in England, Wales, and Northern Ireland for eligible patients ages 12 and older.

That authorization came as the U.K.’s National Institute for Health and Care Excellence (NICE) recommended Orladeyo — marketed by BioCryst Pharmaceuticals — as a treatment for people with HAE who have at least two swelling attacks per month.

The Scottish Medicines Consortium (SMC) is expected to issue a decision on Orladeyo’s use for patients in Scotland under the U.K.’s National Health Service (NHS) in the first half of next year.

“We are excited for HAE patients that this recommendation from NICE provides access to the first oral, once-daily treatment for UK patients to achieve symptom control and experience relief from the burdens of HAE,” Charlie Gayer, chief commercial officer of BioCryst, said in a press release.

Gayer added that NICE’s decision “expands access to modern prophylaxis [preventive treatment] with Orladeyo, compared to the attack frequency requirements from NICE for injectable options.”

Orladeyo was approved by the European Commission in April, and the U.K.’s Medicines and Healthcare products Regulatory Agency issued a similar approval the following month.

It also has been approved in the U.S. and Japan, and is being reviewed by regulatory authorities in Canada, Switzerland, and Israel.

These approvals, and NICE’s recommendation, were supported by data from the Phase 3 APeX-2 trial (NCT03485911), sponsored by BioCryst, in which 121 people with HAE were given Orladeyo or a placebo for about six months.

Results showed that treatment with the medication sustainably reduced the mean rate of monthly swelling attacks by approximately 80% over the course of nearly two years (96 weeks).

Orladeyo also is being evaluated in an open-label Phase 2/3 trial, called APeX-S (NCT03472040). Interim data from this trial have supported the medication’s favorable safety profile, and also showcased its ability to lower the rate of swelling attacks among patients.

Laura Szutowicz, CEO of the charity organization HAE U.K., said her advocacy group “welcomes the NICE decision on berotralstat, which means that eligible patients and clinicians have another choice of treatment for this lifelong condition.”

“The NICE recommendation of berotralstat is great news for clinicians and eligible patients who now have access to the first oral preventive treatment for HAE that could significantly reduce the number of attacks and may improve quality of life,” said Sorena Kiani, PhD, a consultant immunologist at Barts Health NHS Trust.

quote:

Wijze man schreef op 21 september 2021 13:19:

[...]

We zouden toch weer overgaan op Pharming????
Ik snap echt helemaal niets van jou.

quote:

DE MONITOR - Dé Pharming Expert schreef op 21 september 2021 13:14:

[...]

Nu pas..

quote:

DE MONITOR - Dé Pharming Expert schreef op 21 september 2021 13:11:

Oral Orladeyo Now Available in UK to Treat HAE Swelling Attacks

by Marisa Wexler MS | September 20, 2021

oral Orladeyo now available in UK | Angioedema News | woman with megaphone announcement illustration
Orladeyo (berotralstat), a daily oral therapy to prevent swelling attacks in people with hereditary angioedema (HAE), is now available in England, Wales, and Northern Ireland for eligible patients ages 12 and older.

That authorization came as the U.K.’s National Institute for Health and Care Excellence (NICE) recommended Orladeyo — marketed by BioCryst Pharmaceuticals — as a treatment for people with HAE who have at least two swelling attacks per month.

The Scottish Medicines Consortium (SMC) is expected to issue a decision on Orladeyo’s use for patients in Scotland under the U.K.’s National Health Service (NHS) in the first half of next year.

“We are excited for HAE patients that this recommendation from NICE provides access to the first oral, once-daily treatment for UK patients to achieve symptom control and experience relief from the burdens of HAE,” Charlie Gayer, chief commercial officer of BioCryst, said in a press release.

Gayer added that NICE’s decision “expands access to modern prophylaxis [preventive treatment] with Orladeyo, compared to the attack frequency requirements from NICE for injectable options.”

Orladeyo was approved by the European Commission in April, and the U.K.’s Medicines and Healthcare products Regulatory Agency issued a similar approval the following month.

It also has been approved in the U.S. and Japan, and is being reviewed by regulatory authorities in Canada, Switzerland, and Israel.

These approvals, and NICE’s recommendation, were supported by data from the Phase 3 APeX-2 trial (NCT03485911), sponsored by BioCryst, in which 121 people with HAE were given Orladeyo or a placebo for about six months.

Results showed that treatment with the medication sustainably reduced the mean rate of monthly swelling attacks by approximately 80% over the course of nearly two years (96 weeks).

Orladeyo also is being evaluated in an open-label Phase 2/3 trial, called APeX-S (NCT03472040). Interim data from this trial have supported the medication’s favorable safety profile, and also showcased its ability to lower the rate of swelling attacks among patients.

Laura Szutowicz, CEO of the charity organization HAE U.K., said her advocacy group “welcomes the NICE decision on berotralstat, which means that eligible patients and clinicians have another choice of treatment for this lifelong condition.”

“The NICE recommendation of berotralstat is great news for clinicians and eligible patients who now have access to the first oral preventive treatment for HAE that could significantly reduce the number of attacks and may improve quality of life,” said Sorena Kiani, PhD, a consultant immunologist at Barts Health NHS Trust.

quote:

Wijze man schreef op 21 september 2021 13:32:

[...]

www.ema.europa.eu/en/documents/produc...

Had je deze al eens doorgenomen?

quote:

Wijze man schreef op 21 september 2021 13:17:

[Modbreak IEX: Gelieve hier geen persoonlijke vetes uit te vechten, een aantal berichten is bij dezen verwijderd. Waarschuwing.]

quote:

valeo 325 schreef op 21 september 2021 13:22:

DE MONITOR - Dé Pharming Expert
Je zal maar je hele leven wijden aan Pharming
Omdat je waarschijnlijk ooit een keer veel geld ben verloren aan Pharming
Maar ga vooral door met deze nuttige besteding van de dag