Aandeel Pharming Group AEX:PHARM.NL, NL0010391025

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Trebor schreef op 8 september 2022 17:12:

Hoe groen wil je het hebben op een gewone donderdag.
Pharming gaat geweldig. Elke nieuwe dag spannender.
Pharming Go

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Jinxter schreef op 8 september 2022 17:36:

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Nee joh! Forse belediging vind je niet?

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Berendbotje schreef op 8 september 2022 17:31:

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Ze noemde leniolisib zelfs een ‘gap-filler’.
Het werd de enige laatste redding zelfs.

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Rikos schreef op 8 september 2022 17:28:

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U kunt beter reageren op de vraag of u aub de pim veklaring van de ema en/of fda hier te plaatsen.
Tot die tijd verklaart u met grote regelmaat zeker als het gaat over de pim verklaring en de daarbij door u verzonnen goedkeuring om het medicijn, naar uw idee, zonder goedkeuring te mogen verkopen.

In afwachting vannuw reactie verblijven wij,
De realisten.

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Marrr schreef op 8 september 2022 17:36:

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Bij de meesten zie ik wel een plaatje voor me! ;-)

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Jinxter schreef op 8 september 2022 17:35:

Let’s Rock & Roll

Amerika, Amerika
Drie maal in de rondte van je hopsasa

Cheers to Pharming

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jakhals schreef op 8 september 2022 17:34:

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De realiteit is dat we vandaag weer een dikke plus zien op de borden.

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Sharen schreef op 8 september 2022 17:41:

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$.. Nóóóóóiiiit kwam Berend Botje meer om ..$

En wéér 3,5 % erbij…. hoppa

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Rikos schreef op 8 september 2022 17:42:

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Haha, jazekers..... maar wordt alleen ontzettend moe van de fantasie van Mmdeugen.

Het totaal plaatje ziet er inderdaad positief uit.

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Jinxter schreef op 8 september 2022 17:43:

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Hahaha wat een afgang moet dit zijn?

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Jinxter schreef op 8 september 2022 17:40:

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Ik lig helemaal dubbel als je het voor je zie…
Wat een muppetshow zal dat zijn….

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Mmdeugen schreef op 8 september 2022 17:39:

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Beste Rikos,

Ik heb van u nog nooit een antwoord ontvangen op mijn aan u gestelde vragen. U neemt mij niet serieus, dus dus.....

Die PIM goedkeuring kunt u als realisten vinden op de website van Pharming. Febr. 22.

Ook alle realisten heb ik uitgenodigd te reageren op mijn reactie op de PIM, met de vraag of het het goed lees, dat met de PIM goedkeuring van de EMA in februari 2022,
Pharming op enig moment al Leniolisib mag verkopen zonder dat het geneesmiddel is goedgekeurd?

Ik heb er nog geen antwoord op gehad van een van de realisten? En u in het bijzonder!!

Waarom zou er eigenlijk een PIM - mogelijkheid worden geboden, denkt u?

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JET-moon schreef op 8 september 2022 17:16:

gelukkig zijn hier allemaal beleggings experts over de stoplossen. :)

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Rikos schreef op 8 september 2022 17:42:

[...]

Haha, jazekers..... maar wordt alleen ontzettend moe van de fantasie van Mmdeugen.

Het totaal plaatje ziet er inderdaad positief uit.

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Mmdeugen schreef op 8 september 2022 17:20:

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En dan ga je ook nog m.i. blind mee in een " fabeltje" van Bassie!!

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Mmdeugen schreef op 8 september 2022 17:39:

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Pharming op enig moment al Leniolisib mag verkopen zonder dat het geneesmiddel is goedgekeurd?

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Geldding schreef op 8 september 2022 17:48:

PR Newswire: news distribution, targeting and monitoring
Pharming receives agreement of Paediatric Investigation Plan and Promising Innovative Medicine designation for leniolisib from UK MHRA
Pharming Group N.V. logo (PRNewsfoto/Pharming Group N.V.)
NEWS PROVIDED BY

Pharming Group N.V.
Apr 26, 2022, 01:00 ET

An agreed Paediatric Investigation Plan (PIP) is the regulatory pathway to market authorization for leniolisib as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in children

A Promising Innovative Medicine (PIM) designation is an early indication that leniolisib is a candidate for the MHRA's Early Access to Medicines Scheme

LEIDEN, Netherlands, April 26, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or the "Company") (EURONEXT Amsterdam: PHARM) ( Nasdaq: PHAR) announces that it has received a positive decision from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) on a Paediatric Investigation Plan (PIP) submission for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor, for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in patients from 1 year of age to less than 18 years of age. The Company also announces that the MHRA has granted Promising Innovative Medicine (PIM) designation to leniolisib for the treatment of APDS.

A PIP is a development plan aimed at ensuring that the necessary data are obtained to support the marketing authorization of a medicine in the pediatric population. All applications for marketing authorization for new medicines in children require the results of studies as described in an agreed PIP, unless the medicine is exempt due to a deferral or waiver. The leniolisib PIP includes two planned global clinical trials in pediatric patients with APDS, the first in children ages 4-11 years and the second in children ages 1-6 years. The Company expects to initiate recruitment for this pediatric program for leniolisib during the second half of 2022.

A PIM designation indicates that a medicinal product is a promising candidate for the MHRA's Early Access to Medicines Scheme (EAMS), which provides pre-market access to products that are intended for the treatment, diagnosis, or prevention of a life-threatening or seriously debilitating condition and have the potential to address an unmet medical need.

Anurag Relan MD, Pharming's Chief Medical Officer, commented:

"We are pleased to have received agreement of this PIP and the PIM designation from the MHRA, these represent important regulatory milestones as we continue to advance leniolisib for the treatment of APDS, a rare, often debilitating, and sometimes fatal condition. The agreed PIP and the PIM designation further support our confidence in the potential of leniolisib to address APDS, as they provide us with a pathway toward marketing authorization in the United Kingdom. Pharming looks forward to continuing to work with regulatory authorities globally to bring leniolisib to patients with this significant unmet medical need as expeditiously as possible."

Leniolisib, which has received Orphan Drug Designation in Europe, received the same designation from the US Food and Drug Administration on January 30, 2018.

As previously announced, Pharming plans to begin submitting global registration filings for leniolisib in the second quarter of 2022 and, subject to approval, launching the treatment in the US in the first quarter of 2023 and starting a series of European launches in the second half of 2023.

About Activated Phosphoinositide 3-Kinase d Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately one to two people per million. Also known as PASLI, it is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kd (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3Kd pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.

About leniolisib

Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger specifically activating AKT and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Ka and PI3Kß, which are ubiquitously expressed, PI3K? and PI3K? are expressed primarily in cells of hematopoietic origin. The central role of PI3K? in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3K? is a valid and potentially effective therapeutic target for several immune diseases.

To date, leniolisib has been well tolerated during both a Phase 1 first-in-human trial in healthy subjects and a Phase II/III registration-enabling study.

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